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 65. 原発性免疫不全症候群 [臨床試験数:273,薬物数:388(DrugBank:87),標的遺伝子数:66,標的パスウェイ数:57] 

Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
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agemin
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PhaseCountries
1NCT04154488December 11, 201911 November 2019A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Neutropenia DisordersA Phase 1B, Open-Label, Multicenter Study of Mavorixafor in Patients With Severe Congenital Neutropenia and Chronic Neutropenia DisordersNeutropeniaDrug: MavorixaforX4 PharmaceuticalsNot recruiting12 YearsN/AAll45Phase 1United States
2NCT03814798December 201922 July 2019Study Evaluating IGSC 20% Flexible Dosing in Treatment-Experienced and Treatment-Naive Subjects With Primary ImmunodeficiencyA Multicenter, Randomized, Cross-over, Open-label Study to Evaluate IGSC 20% Flexible Dosing Including Daily Push Dosing In Treatment-Experienced Subjects With Primary Immunodeficiency (PI) and Evaluation of Loading/Maintenance IGSC 20% in Treatment-Naïve Subjects With PIPrimary ImmunodeficiencyBiological: IGSC 20% daily push versus every 2 weeks pump;Biological: IGSC 20% daily push versus once a week pump;Biological: IGSC 20% daily push versus 2 times per week pump;Biological: IGSC 20% 150 mg/kgGrifols Therapeutics LLCNot recruiting2 Years75 YearsAll60Phase 3United States
3NCT04140539November 11, 20194 November 2019A Clinical Study to Enable Process Validation of Commercial Grade OTL-101A Single Arm, Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hematopoietic Stem Cell Gene Therapy (OTL-101) in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)Severe Combined Immunodeficiency Due to ADA DeficiencyBiological: OTL-101Orchard TherapeuticsUniversity of California, Los AngelesRecruitingN/A17 YearsAll3Phase 2/Phase 3United States
4NCT03597594November 201911 November 2019Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)Severe Combined ImmunodeficiencyDrug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte InfusionSt. Jude Children's Research HospitalNot recruiting2 MonthsN/AAll42Phase 1/Phase 2United States
5NCT03896932November 20198 April 2019Minipooled-IVIG in Primary Immunodeficiency DiseaseStudy of Safety and Efficacy of Mini-pool Intravenous Immunoglobulin (MP-IVIG) Prepared by Assiut University Hospital Blood Bank in Primary Immunodeficiency PatientsPrimary ImmunodeficiencyOther: minipooled- Intravenous immunoglobulin(MP-IVIG)Assiut UniversityNot recruitingN/A18 YearsAll15N/A
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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PhaseCountries
6NCT03910452October 28, 201911 November 2019Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cellNational Institute of Allergy and Infectious Diseases (NIAID)Recruiting4 Years65 YearsAll30Early Phase 1United States
7NCT03995108October 17, 20194 November 2019Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) SyndromeA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label ExtensionWHIM SyndromeDrug: Mavorixafor;Drug: PlaceboX4 PharmaceuticalsRecruiting12 YearsN/AAll28Phase 3United States
8NCT04049084September 26, 20194 November 2019An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCIDAn Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency (SCID)Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)Orchard TherapeuticsUniversity of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation TrustRecruitingN/AN/AAll70Phase 2United States;United Kingdom
9NCT02860559September 201923 April 2019Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined ImmunodeficiencySafety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined ImmunodeficiencySevere Combined ImmunodeficiencyBiological: TBX-1400Taiga Biotechnologies, Inc.Not recruiting1 Month4 YearsAll8Phase 1Israel
10NCT03939533September 201930 September 2019Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency DiseasesClinical Phase 3 Study to Monitor the Safety, Tolerability, and Efficacy of Subcutaneous Human Immunoglobulin (CUTAQUIG®) Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency DiseasesPrimary Immune Deficiency DisorderDrug: CUTAQUIGOctapharmaNot recruiting2 Years75 YearsAll65Phase 3
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
11NCT03974178September 201926 August 2019Efficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei RhodesienseEfficacy and Safety of Fexinidazole in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Rhodesiense: a Multicentre, Open-label Clinical TrialTrypanosoma Brucei Rhodesiense; InfectionDrug: FexinidazoleDrugs for Neglected DiseasesEuropean and Developing Countries Clinical Trials Partnership (EDCTP)Not recruiting6 YearsN/AAll50Phase 2/Phase 3
12NCT03984890August 1, 20194 November 2019Vitamin D3 For CGD Patients With BCGosis/ItisEffect of Vitamin D3 Supplementation on Chronic Granulomatous Disease Patients With BCGosis/ItisVitamin D3;Chronic-granulomatous Disease;BCGDrug: Traditional treatment of CGD and TB;Drug: Vitamin D3Children's Hospital of Fudan UniversityRecruitingN/A18 YearsAll50Phase 2/Phase 3China
13NCT03878069June 201925 March 2019Registry Study of Revcovi Treatment in Patients With ADA-SCIDSingle Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement TherapyAdenosine Deaminase Deficiency;Severe Combined ImmunodeficiencyBiological: elapegademase-lvlrLeadiant Biosciences, Inc.Not recruitingN/A65 YearsAll20N/A
14NCT03961009April 30, 20193 June 2019Efficacy, Safety and Pharmacokinetics Study of Kedrion IVIG 10% to Treat Participants With Primary Immunodeficiency (PID)A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety and Pharmacokinetics of Kedrion Intravenous Immunoglobulin (IVIg) 10% in Primary Immunodeficiency Disease (PID) PatientsPrimary Immunodeficiency DiseaseBiological: Kedrion IVIG 10%Kedrion S.p.A.Recruiting2 Years70 YearsAll50Phase 3United States;Canada
15NCT03825783April 15, 20199 September 2019A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-IGene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene.Leukocyte Adhesion Defect - Type IBiological: RP-L201Rocket Pharmaceuticals Inc.Recruiting3 MonthsN/AAll2Phase 1Spain
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
16NCT03547830April 13, 201930 September 2019Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD PatientsA Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Plerixafor;Drug: GcsfFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyRecruiting1 Month24 YearsAll17Phase 2Russian Federation
17NCT03601286December 21, 201829 April 2019Lentiviral Gene Therapy for X-linked Severe Combined ImmunodeficiencyPhase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted BusulfanSevere Combined Immunodeficiency, X-LinkedDrug: Lentiviral vector transduced CD34+ cellsGreat Ormond Street Hospital for Children NHS Foundation TrustRecruiting8 Weeks5 YearsMale5Phase 1United Kingdom
18NCT03812263November 30, 20184 February 2019A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-IGene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 GeneLeukocyte Adhesion Defect - Type IBiological: RP-L201Rocket Pharmaceuticals Inc.Recruiting3 MonthsN/AAll9Phase 1/Phase 2United States
19EUCTR2018-002680-26-ES27/11/20187 January 2019Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 GeneGene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector Encoding the ITGB2 GeneLeukocyte Adhesion Deficiency-I (LAD-I)
MedDRA version: 20.0 Level: LLT Classification code 10018137 Term: Genetic anomalies of leukocytes System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: LADICell
Product Code: RP-L201
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: CD34+CELLS
Other descriptive name: CD34+CELLS
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: not less then
Concentration number: 4000000-
Rocket Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
2Phase 1Spain
20EUCTR2018-001029-14-FR25/10/20186 November 2018HTLPA phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients - HTLP NECKERSCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT.
MedDRA version: 20.0 Level: PT Classification code 10010099 Term: Combined immunodeficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: HTLP
Product Code: HTLP
Pharmaceutical Form: Suspension for injection
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)AuthorisedFemale: yes
Male: yes
12Phase 1;Phase 2France
No.TrialIDDate_
enrollement
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PhaseCountries
21NCT03619551October 22, 201824 June 2019Conditioning SCID Infants Diagnosed EarlyA Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRaß+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC)SCIDDrug: Busulfan;Device: Cell processing for TCRaß+/CD19+ depletionMichael Pulsipher, MDRecruitingN/A2 YearsAll64Phase 2United States
22NCT03716700September 24, 20183 December 2018Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN)Real-world CANadian CUvitru Non-Interventional Study in Subjects Transitioning From Subcutaneous Immunoglobulin (CANCUN)Primary Immunodeficiency Diseases (PID)Biological: CUVITRUBaxalta now part of ShireBaxalta Innovations GmbH, now part of Shire;Baxalta US Inc., now part of ShireRecruiting3 YearsN/AAll500N/ACanada
23EUCTR2018-000338-36-DE20/09/201826 November 2018Not ApplicableOpen-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDATPatient with neurological symptoms of Ataxia Telangiectasia
MedDRA version: 20.1 Level: PT Classification code 10003594 Term: Ataxia telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Dexamethasone sodium phosphate
Pharmaceutical Form: Solution for blood fraction modification
INN or Proposed INN: Dexamethasone sodium phosphate
CAS Number: 2392-39-4
Current Sponsor code: DSP
Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR.
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
EryDel S.p.A.AuthorisedFemale: yes
Male: yes
51Phase 3United States;Poland;Belgium;Spain;Australia;Israel;Germany;India
24NCT03663933September 4, 20184 November 2019Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or DysregulationPhase II Trial of Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or DysregulationLymphoproliferative Disorders;Autoimmune Lymphoproliferative;Primary T-cell Immunodeficiency Disorders;Immune System Diseases;Common Variable ImmunodeficiencyDrug: Immunosuppression Only Conditioning (IOC);Drug: Reduced Intensity Conditioning (RIC);Drug: GVHD Prophylaxis;Procedure: Allogeneic HSCNational Cancer Institute (NCI)Recruiting4 YearsN/AAll177Phase 2United States
25NCT03907241September 1, 201824 June 2019Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases, Including (But Not Limited to) Those Who Have Completed the SCGAM-01 TrialClinical Phase III Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases, Including (But Not Limited to) Those Who Have Completed the SCGAM-01 TrialPrimary ImmunodeficiencyDrug: Octanorm 16.5%OctapharmaNot recruiting18 Years75 YearsAll6Phase 3Canada
No.TrialIDDate_
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PhaseCountries
26EUCTR2018-001489-41-SE02/07/201823 July 2018A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis congenita / telomere disease, inherited genetic conditions.Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere diseaseDyskeratosis congenita / telomere disease
MedDRA version: 20.0 Level: PT Classification code 10001756 Term: Allogenic bone marrow transplantation therapy System Organ Class: 10042613 - Surgical and medical procedures ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Lemtrada
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Alemtuzumab
CAS Number: 216503-57-0
Other descriptive name: ALEMTUZUMAB
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0,2-
Trade Name: Fludarabine
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: FLUDARABINE
CAS Number: 21679-14-1
Concentration unit: mg/m2 milligram(s)/square meter
Concentration type: equal
Concentration number: 30-
Karolinska UniversitetssjukhusetAuthorisedFemale: yes
Male: yes
20Phase 2United States;Sweden
27NCT03366142July 2, 201828 October 2019Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory PathologyUse of Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory PathologyLAD1Drug: UstekinumabNational Institute of Allergy and Infectious Diseases (NIAID)Recruiting12 Years65 YearsAll20Phase 1/Phase 2United States
28NCT03513328June 15, 20183 June 2019Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchRecruiting6 Months38 YearsAll40Phase 1/Phase 2United States
29NCT03576469June 13, 201826 August 2019A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG InfusionsA Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG InfusionsCVI - Common Variable ImmunodeficiencyBiological: C1-esterase inhibitor [recombinant] (C1-INH-R)IMMUNOe Research CentersRecruiting18 YearsN/AAll20Phase 4United States
30EUCTR2018-000338-36-ES31/05/201818 June 2018Not ApplicableOpen-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDATPatient with neurological symptoms of Ataxia Telangiectasia
MedDRA version: 20.1 Level: PT Classification code 10003594 Term: Ataxia telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Dexamethasone sodium phosphate
Pharmaceutical Form: Solution for blood fraction modification
INN or Proposed INN: Dexamethasone sodium phosphate
CAS Number: 2392-39-4
Current Sponsor code: DSP
Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR.
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
EryDel S.p.A.AuthorisedFemale: yes
Male: yes
61Phase 3United States;Poland;Belgium;Spain;Australia;Israel;Norway;Germany;Italy;United Kingdom;India
No.TrialIDDate_
enrollement
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31NCT03538899May 31, 201815 July 2019Autologous Gene Therapy for Artemis-Deficient SCIDA Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic CellsSevere Combined ImmunodeficiencyDrug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: BusulfanUniversity of California, San FranciscoRecruiting2 MonthsN/AAll15Phase 1/Phase 2United States
32NCT03548818May 16, 201815 July 2019Role of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous DiseaseRole of Interferon-gamma 1-b (IFN-?) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Interferon Gamma-1BUniversity of Colorado, DenverRecruiting5 Years60 YearsAll20Phase 3United States
33NCT03232203April 12, 201827 May 2019Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ TreatmentSevere Combined Immunodeficiency Due to ADA DeficiencyDrug: STRIMVELISOrchard TherapeuticsRecruiting18 YearsN/AAll10N/AItaly
34NCT03512314January 24, 201826 August 2019Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label ExtensionOpen-label Extension Study With Tadekinig Alfa (r-hIL-18BP) to Monitor Safety and Tolerability in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP DeficiencyXIAP Deficiency;NLRC4-MASDrug: Tadekinig alfaAB2 Bio Ltd.RecruitingN/AN/AAll10Phase 3United States;Canada;Germany
35NCT03311503January 19, 201811 February 2019Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningPhase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningSevere Combined Immunodeficiency, X Linked;Gene TherapyBiological: autologous CD34+ cell transduced with G2SCID vectorDavid WilliamsUniversity of California, Los AngelesRecruitingN/A5 YearsMale10Phase 1/Phase 2United States;United Kingdom
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36JPRN-UMIN0000308062018/01/172 April 2019A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich SyndromeWiskott-Aldrich syndromeWASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan.
1. Rituximab (day-22)
375 mg/m2

2. Preconditioning chemotherapy
Fludarabine 30mg/m2 x 2 (day-3, day-2)
Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours)

3. Infusion of WASP cDNA-transduced CD34 positive HSC
5 x 10^6/kg (at least 3 x 10^6/kg)
National Center for Child Heath and DevelopmentRecruitingNot applicableNot applicableMale3Phase 1,2Japan
37JPRN-UMIN0000306472017/12/302 April 2019Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial (CGD-RIST2)Chronic granulomatous diseaseConditioning regimen with targeted-busulfan and fludarabin
Total body irradiation (3Gy) at day -1
Stem cell transplantation at day 0
National Center for Child Health and DevelopmentRecruitingNot applicable25years-oldMale and Female22Not selectedJapan
38NCT03333486December 7, 201725 June 2018Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Recruiting1 Year75 YearsAll58Phase 2United States
39NCT03330795December 1, 201714 October 2019Bilateral Orthotopic Lung Transplant - Bone Marrow TransplantBilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA Matched Cadaveric Donors (RTB-003)Primary Immunodeficiency;PIDBiological: CD3/CD19 neg allogeneic BMTNational Institute of Allergy and Infectious Diseases (NIAID)University of PittsburghRecruiting10 Years45 YearsAll8Phase 1/Phase 2United States
40JPRN-UMIN0000293242017/09/2823 April 2019Double-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitisDouble-blind placebo-controlled clinical trial of thalidomide for chronic granulomatous disease-associated colitis - Clinical trial of thalidomide for CGD colitisChronic granulomatous disease associated colitisOral administration of thalidomide (1.5-3mg/kg), once a day
Oral administration of placebo (1.5-3mg/kg), once a day
National Center for Child Heath and DevelopmentRecruiting1years-oldNot applicableMale and Female8Not selectedJapan
No.TrialIDDate_
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41NCT03277313September 25, 20172 September 2019Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD SubjectsEfficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: HYQVIA;Biological: GAMMAGARD LIQUIDBaxalta now part of ShireBaxalta Innovations GmbH, now part of ShireNot recruiting2 Years15 YearsAll44Phase 3United States
42NCT03080480September 1, 20174 November 2019Pioglitazone Therapy for Chronic Granulomatous DiseaseEfficacy and Safety of Pioglitazone Therapy for Chronic Granulomatous Disease Patients With Severe Infection.Chronic Granulomatous DiseaseDrug: PioglitazoneChildren's Hospital of Fudan UniversityNot recruiting1 Month18 YearsAll3Phase 1/Phase 2China
43NCT03113760July 21, 201730 September 2019Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP DeficiencyMulticenter, Double-blind, Placebo-controlled, Randomized Withdrawal Trial With Tadekinig Alfa (r-hIL-18BP) in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP DeficiencyNLRC4-MAS;XIAP DeficiencyDrug: Tadekinig alfa;Other: 0.9% sodium chlorideAB2 Bio Ltd.RecruitingN/A17 YearsAll10Phase 3United States;Canada;Germany
44NCT03217617July 15, 201730 September 2019Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)Gene Transfer for X-linked Severe Combined Immunodeficiency (SCID-X1) Using a Self-inactivating Lentiviral Vector (TYF-IL-2Rg)SCID, X LinkedBiological: TYF-IL-2Rg gene-modified autologous stem cellsShenzhen Geno-Immune Medical InstituteRecruiting1 Month10 YearsMale10Phase 1/Phase 2China
45NCT03116347June 13, 201726 August 2019Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD SubjectsPost-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: HYQVIA;Biological: KIOVIG;Biological: CuvitruBaxalta now part of ShireBaxalta Innovations GmbH, now part of ShireRecruiting2 Years17 YearsAll40Phase 4Czechia;Denmark;France;Greece;Slovakia;Sweden;United Kingdom
No.TrialIDDate_
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46NCT03988426March 7, 20171 July 2019Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency DiseasesClinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases.Primary Immune Deficiency DisorderBiological: OctanormOctapharmaNot recruiting18 Years70 YearsAll25Phase 3Russian Federation
47NCT03112655February 24, 201730 September 2019Diagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: Early Test-of-cureDiagnostic Tools for Human African Trypanosomiasis Elimination and Clinical Trials: WP4 Early Test-of-cureAfrican Trypanosomiasis;African; Trypanosomiasis, West;Sleeping Sickness; West African;Trypanosoma Brucei Gambiense; InfectionDiagnostic Test: RNA and neopterin detectionInstitut de Recherche pour le DeveloppementInstitute of Tropical Medicine, Belgium;Institut National de Recherche Biomédicale. Kinshasa, République Démocratique du Congo;Ministry of Public Health, Democratic Republic of the CongoNot recruiting15 YearsN/AAll88N/ACongo, The Democratic Republic of the
48EUCTR2016-003438-26-GB14/02/201719 November 2018Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study.Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency DiseasesPrimary Immunodeficiency Disease (PIDD)
MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use
Product Name: HyQvia
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Trade Name: KIOVIG 100 mg/ml solution for infusion
Product Name: KIOVIG
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection
Product Name: Cuvitru
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human Normal Immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Baxalta US Inc.AuthorisedFemale: yes
Male: yes
40Phase 4France;Czech Republic;Slovakia;Greece;Denmark;United Kingdom;Sweden
49NCT03033745February 1, 201714 January 2019Safety and Tolerability of Higher Infusion Parameters of IgPro20 (Hizentra) in Subjects With Primary Immunodeficiency (PID)An Open-label Multicenter Study to Evaluate the Safety and Tolerability of Higher Infusion Parameters of Immune Globulin Subcutaneous (Human), 20% Liquid (Hizentra®) in Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyDrug: IgPro20CSL BehringNot recruiting2 YearsN/AAll49Phase 4United States;Canada
50NCT03733249January 201728 October 2019Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyFollow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological DisordersAcute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle CellDrug: Rimiducid;Biological: rivogenlecleucelBellicum PharmaceuticalsRecruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;Saudi Arabia;United Kingdom
No.TrialIDDate_
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51NCT03054181December 22, 20164 March 2019Facilitated Immunoglobulin Administration Registry and Outcomes Study (FIGARO)Facilitated Immunoglobulin Administration Registry and Outcomes StudyPrimary Immunodeficiency;Secondary Immune DeficiencyBiological: HyQviaGWT-TUD GmbHRecruitingN/AN/AAll100Phase 3France;Germany;Italy
52NCT03005327December 201615 July 2019A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) SyndromeA Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM SyndromeWHIM SyndromeDrug: X4P-001X4 PharmaceuticalsNot recruiting18 YearsN/AAll15Phase 2United States;Australia
53NCT03037359December 201616 December 2017A Study About Low Blood Pressure in Patients With Primary Immunodeficiency Disease Treated With Immune Globulin ProductsA Multicenter, Non-interventional, Observational, Prospective Study to Assess Hypotension in Patients With Primary Immunodeficiency Disease Treated With Bivigam™ (Human 10%) or Other Commercial Human 10% Immune Globulin (Intravenous) (IGIV) Products During Infusion and up to 72 Hours Post InfusionPrimary Immune Deficiency DisorderBiological: Bivigam;Biological: OtherBiotest Pharmaceuticals CorporationRecruitingN/AN/AAll200N/AUnited States
54NCT03025789November 17, 201620 May 2019Fexinidazole in Human African Trypanosomiasis Due to T.b. Gambiense at Any StageAn Open-label Study Assessing Effectiveness, Safety and Compliance With Fexinidazole in Patients With Human African Trypanosomiasis Due to T.b. Gambiense at Any StageTrypanosomiasis, African;Sleeping Sickness;Trypanosomiasis; GambianDrug: FexinidazoleDrugs for Neglected DiseasesSanofiRecruiting6 YearsN/AAll174Phase 3Congo, The Democratic Republic of the;Guinea
55NCT02881437November 11, 201615 July 2019IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQviaAssessment of the IgG Trough Level in Subjects With Primary Immunodeficiency Switching From Standard Subcutaneous Immunoglobulin (SCIG) to Every Other Week HyQviaPrimary ImmunodeficiencyDrug: IgHy10University Hospital, LilleShireNot recruiting18 YearsN/AAll22Phase 4France
No.TrialIDDate_
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56NCT03087955October 11, 201619 November 2018Prospective Study on Efficacy and Safety of SCYX-7158 in Patients Infected by Human African Trypanosomiasis Due to T.b. GambienseEfficacy and Safety Study of SCYX-7158 in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective StudyTrypanosomiasis, African;Gambiense Trypanosomiasis;Sleeping SicknessDrug: SCYX 7158Drugs for Neglected DiseasesRecruiting15 YearsN/AAll360Phase 2/Phase 3Congo, The Democratic Republic of the;Guinea
57EUCTR2015-003652-52-DE29/09/201614 May 2018An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID)An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID)Replacement therapy in patients with primary immunodeficiency disease (PID)
MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: IgG Next Generation
Product Code: BT595
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Current Sponsor code: BT595
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Biotest AGAuthorisedFemale: yes
Male: yes
70Phase 3United States;Spain;Russian Federation;Germany;United Kingdom
58EUCTR2015-005241-31-DE14/09/201626 November 2018n/aA Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTESTPatient with neurological symptoms of Ataxia Telangiectasia
MedDRA version: 20.1 Level: PT Classification code 10003594 Term: Ataxia telangiectasia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Dexamethasone sodium phosphate
Pharmaceutical Form: Solution for blood fraction modification
INN or Proposed INN: Dexamethasone sodium phosphate
Current Sponsor code: DSP
Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR.
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intravenous use
EryDel S.p.A.AuthorisedFemale: yes
Male: yes
180Phase 3United States;Spain;Costa Rica;Turkey;Israel;United Kingdom;Italy;India;Poland;Belgium;Australia;Germany;Tunisia;Norway
59NCT01512888August 17, 201625 February 2019Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed InfantsA Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic CellsSevere Combined Immunodeficiency Disease, X-linkedGenetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacsSt. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM)RecruitingN/A24 MonthsMale28Phase 1/Phase 2United States
60EUCTR2015-003369-27-DE01/07/201611 June 2018A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID)A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID) - REGAIN: REGulatory T cells and Aldesleukin for Immunodeficiency- associated eNteropathyautoimmune enteropathy (AIE) in common variable immunodeficiency (CVID).
MedDRA version: 19.0 Level: PT Classification code 10021449 Term: Immunodeficiency common variable System Organ Class: 10021428 - Immune system disorders
MedDRA version: 19.0 Level: LLT Classification code 10017922 Term: Gastroenteropathy NOS System Organ Class: 100000004856 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: PROLEUKIN® S
Product Name: Aldesleukin
Pharmaceutical Form: Powder for solution for injection/infusion
INN or Proposed INN: Aldesleukin
CAS Number: 110942-02-4
Other descriptive name: ALDESLEUKIN
Concentration unit: IU/ml international unit(s)/millilitre
Concentration type: equal
Concentration number: 18x10 6-
Universitätsklinikum FreiburgNot RecruitingFemale: yes
Male: yes
6Phase 2Germany
No.TrialIDDate_
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61NCT02609932July 201625 February 2019Effect of IFN-? on Innate Immune CellsEffect of Interferon-gamma 1-b on Innate Immune CellsChronic Granulomatous DiseaseDrug: Administration of drug (Interferon-gamma 1-b) subcutaneouslyUniversity of Colorado, DenverNot recruiting18 Years60 YearsAll20Phase 1United States
62NCT02789397July 201611 June 2018Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID)Clinical Trial to Assess the Efficacy of Rituximab and Azathioprine in the Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) in Adult Patients With Common Variable Immunodeficiency (CVID)Granulomatous and Lymphocytic Interstitial Lung DiseaseDrug: Rituximab (RTX) and Azathioprine (AZA);Drug: Placebo AdministrationMedical College of WisconsinNot recruiting18 YearsN/AAll0Phase 2
63NCT02737384June 14, 201611 June 2019Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID)Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID): Selective Depletion of Naive Cells From the GraftCombined ImmunodeficienciesBiological: Depletion in CD45RA graft donorAssistance Publique - Hôpitaux de ParisNot recruiting12 Months18 YearsAll4Phase 2France
64JPRN-UMIN0000226882016/06/102 April 2019A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous diseaseChronic granulomatous diseaseConditioning regimen with targeted-busulfan and fludarabin
Total body irradiation (3Gy) at day -1
Stem cell transplantation at day 0
National Center for Child Health and DevelopmentRecruitingNot applicable25years-oldMale and Female9Not selectedJapan
65NCT02806986June 201622 July 2019Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary ImmunodeficiencyA Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyBiological: IGSC 20%Grifols Therapeutics LLCNot recruiting2 Years75 YearsAll61Phase 3Australia;Czechia;France;Germany;Hungary;Poland;Spain;Sweden;United Kingdom;Czech Republic
No.TrialIDDate_
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66NCT02822495June 201615 April 2019Expanded Access Protocol for Tabelecleucel for Patients With Epstein-Barr Virus-Associated Viremia or MalignanciesExpanded Access Protocol for Providing Tabelecleucel to Patients With Epstein-Barr Virus-Associated Viremia or Malignancies for Whom There Are No Appropriate Alternative TherapiesEpstein-Barr Virus (EBV) Infections;Lymphoproliferative Disorders;EBV+ Associated Lymphoma;EBV+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD);Epstein-Barr Viremia;Lymphoma, AIDS-related;Epstein-Barr Virus-associated Lymphoproliferative Disease (EBV+ LPD) With Primary Immunodeficiency (PID);Leiomyosarcoma (LMS);Nasopharyngeal Carcinoma (NPC);Epstein-Barr Virus-associated Lymphoproliferative Disease (EBV+ LPD) With Acquired Immunodeficiency (AID);Solid Organ Transplant Complications;Stem Cell Transplant ComplicationsBiological: tabelecleucelAtara BiotherapeuticsNot recruitingN/AN/AAllPhase 1/Phase 2United States
67NCT03019809June 201618 December 2018A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS PatientsA Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.Wiskott-Aldrich Syndrome;Hematopoietic Stem Cell Transplantation;Graft FailureBiological: Plerixafor for Conditioning before HSCT.;Biological: G-CSF for Conditioning before HSCT.Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyRecruiting1 Month19 YearsAll30Phase 2Russian Federation
68EUCTR2015-003290-15-GB12/05/201622 May 2017A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary ImmunodeficiencyA Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency - GTI1503Primary Immunodeficiency (PI) diseases
MedDRA version: 20.0 Level: PT Classification code 10061598 Term: Immunodeficiency System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.0 Level: LLT Classification code 10045792 Term: Unspecified disorder of immune mechanism System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12]
Product Name: Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%)
Product Code: IGSC 20%
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified
Current Sponsor code: GRF6017
Concentration unit: % percent
Concentration type: equal
Concentration number: 20-
Grifols Therapeutics Inc.AuthorisedFemale: yes
Male: yes
60Phase 3France;Hungary;Czech Republic;Poland;Spain;Australia;Germany;United Kingdom;Sweden
69EUCTR2015-002491-24-DE11/05/20167 January 2019Safety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related diseaseSafety of Abatacept in patients with interstitial lung disease and common variable immunodeficiency (CVID) and related disease - SAILCVID patients confirmed according to ESID/PAGID criteria or related disorders which fulfill the diagnostic criteria for CVID and interstitial lung disease or granuloma diagnosed by chest CT positive for nodules, lines or ground-glass signs
MedDRA version: 18.1 Level: PT Classification code 10022611 Term: Interstitial lung disease System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 18.1 Level: PT Classification code 10021449 Term: Immunodeficiency common variable System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: ORENCIA® 125 mg solution for injection in pre-filled syringe
Product Name: Abatacept
Product Code: BMS-188667
Pharmaceutical Form: Solution for injection in pre-filled syringe
Universitätsklinikum Freiburg, vertreten durch den Leitenden Ärztlichen DirektorNot RecruitingFemale: yes
Male: yes
10Phase 2Germany
70NCT02627300March 20167 October 2019Study of Octanorm Subcutaneous IG in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 TrialClinical Phase III Study to Monitor the Safety, Tolerability and Efficacy of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases Who Have Completed the SCGAM-01 TrialPrimary Immunodeficiency DiseaseDrug: Octanorm 16.5%OctapharmaNot recruitingN/AN/AAll21Phase 3United States;Canada
No.TrialIDDate_
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71NCT02123615January 20163 August 2015ASIS for GAMMAGARD in Primary ImmunodeficiencyASIS for GAMMAGARD in Primary ImmunodeficiencyPrimary ImmunodeficiencyDrug: Gadolinium For abdomen;Drug: Gadolinium For lower back;Drug: Efficacy of Gammagard subcutaneously at Week 12;Drug: Efficacy of Gammagard subcutaneously at Week 24;Drug: Efficacy of Gammagard subcutaneously at Week 36;Drug: Efficacy of Gammagard subdermally at Week 36;Drug: Efficacy of Gammagard subdermally at Week 12;Drug: Efficacy of Gammagard subdermally at Week 24;Drug: Adverse Reactions of Gammagard subcutaneously at Week 12;Drug: Adverse Reactions of Gammagard subcutaneously at Week 24;Drug: Adverse Reactions of Gammagard subcutaneously at Week 36;Drug: Adverse Reactions of Gammagard subdermally at Week 12;Drug: Adverse Reactions of Gammagard subdermally at Week 24;Drug: Adverse Reactions of Gammagard subdermally at Week 36ASIS CorporationNot recruiting21 Years65 YearsBoth60Phase 1/Phase 2United States
72EUCTR2016-001631-12-Outside-EU/EEA6 February 2017The pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiencyProspective open-label single-arm study of the pharmacokinetics and safety of intravenous IgPro10 in Japanese subjects with primary immunodeficiencyPrimary immunodeficiency
MedDRA version: 19.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Privigen
Product Name: Privigen; immunoglobulin intravenous (human)
Product Code: IgPro10
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
CSL Behring KKNot AvailableFemale: yes
Male: yes
10Phase 3Japan
73EUCTR2015-002001-11-DE22/12/20152 October 2017Study of MEDI4736, when used alone or in combination with Tremelimumab, as second line chemotherapy in metastatic Pancreatic Ductal AdenocarcinomaA Phase II Open-Label, Multi-Center Study of MEDI4736 Evaluated as Single Agent or in Combination with Tremelimumab in Patients with Metastatic Pancreatic Ductal Adenocarcinoma - ALPSAdult patients (aged =18 years) with histologically or cytologically confirmed metastatic PDAC, and had tumor progression following prior standard first-line 5-FU-containing or gemcitabine-containing chemotherapy.
MedDRA version: 18.0 Level: LLT Classification code 10033605 Term: Pancreatic cancer metastatic System Organ Class: 100000004864 ;Therapeutic area: Diseases [C] - Cancer [C04]
Product Name: MEDI4736
Product Code: MEDI4736
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: durvalumab
CAS Number: 1428935-60-7
Current Sponsor code: MEDI4736
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: -50
Product Name: Tremelimumab
Product Code: MEDI1123
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Tremelimumab
CAS Number: 745013-19-6
Current Sponsor code: MEDI1123
Other descriptive name: MEDI1123
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-
AstraZeneca ABNot RecruitingFemale: yes
Male: yes
130Phase 2United States;Canada;Spain;Netherlands;Germany;Japan;Korea, Republic of
74NCT02629120December 10, 201511 November 2019High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseHigh Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseChronic Granulomatous Disease TransplantDrug: Campath;Drug: Busulfan IV;Drug: Sirolimus;Drug: cyclophosphamideNational Institute of Allergy and Infectious Diseases (NIAID)Recruiting4 Years65 YearsAll50Phase 1/Phase 2United States
75NCT02604810December 20151 October 2018Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary ImmunodeficiencyAn Open-label, Multi-center Study to Evaluate the Safety and Pharmacokinetics of IGSC 20% Administered for 6 Months in Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyBiological: IGIV-C 10%;Biological: IGSC 20%Grifols Therapeutics LLCNot recruiting2 Years75 YearsAll53Phase 3United States;Canada
No.TrialIDDate_
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agemin
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PhaseCountries
76NCT02579967November 19, 20152 September 2019Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary ImmunodeficienciesPilot Trial of Allogeneic Blood or Marrow Transplantation for Primary ImmunodeficienciesPrimary T-cell Immunodeficiency Disorders;Common Variable Immunodeficiency;Immune System Diseases;Autoimmune Lymphoproliferative;Lymphoproliferative DisordersDrug: Immunosuppression Only Conditioning - Closed with amendment L;Drug: Reduced Intensity Conditioning;Drug: Myeloablative Conditioning-Closed with amendment L;Drug: GVHD Prophylaxis;Procedure: Allo BMTNational Cancer Institute (NCI)Recruiting4 Years75 YearsAll224Phase 2United States
77NCT02593188November 12, 201525 February 2019Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)Primary Immunodeficiency Diseases (PID)Biological: HYQVIABaxalta now part of ShireRecruiting16 YearsN/AAll250Phase 3United States
78NCT02234934October 29, 201511 November 2019Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous DiseaseA Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous DiseaseGranulomatous Disease, Chronic, X-linkedBiological: Lentiviral G1XCGD Gene TherapyUniversity of California, Los AngelesBoston Children’s Hospital;National Institute of Allergy and Infectious Diseases (NIAID);Genethon;California Institute for Regenerative Medicine (CIRM)Not recruiting23 MonthsN/AMale10Phase 1/Phase 2United States
79EUCTR2015-002356-27-IT16/10/20157 December 2015Evaluation of safety and efficacy of the combination of Ibuprofen (IBU), G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by XCGDA multicentric, exploratory, non-randomised, non-controlled, prospective, open-label phase II, study evaluating safety and efficacy of IBU, G-CSF and Plerixafor as a stem cell mobilization regimen in patients affected by X-CGD.X-linked chronic granulomatous disease
MedDRA version: 18.0 Level: PT Classification code 10008906 Term: Chronic granulomatous disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Ibuprofen
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IBUPROFEN
CAS Number: 15687-27-1
Current Sponsor code: NA
Other descriptive name: NA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Trade Name: MYELOSTIM 34 milions UI/ml, powder and solvent for solution for injection or infusion
Product Name: MYELOSTIM 34 milions UI/ml - powder and solvent for solution for injection/infusion
Pharmaceutical Form: Powder and solvent for solution for injection/infusion
INN or Proposed INN: LENOGRASTIM
CAS Number: 135968-09-1
Concentration unit: U/ml unit(s)/millilitre
Concentration type: equal
Concentration number: 34-
Trade Name: Mozobil 20mg/mL vial (injectable solution for subcutaneous use)
Product Name: Mozobil 20mg/mL vial (injectable solution, subcutaneous use)
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Plerixafor
CAS Number: 110078-46-1
Other descriptive name: Plerixafor
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-
Product Name: Pantoprazolo 20 mg gastro-resistant tablets
Pharmaceutical Form: Gastro-resistant tablet
INN or Proposed INN: Pantoprazole
CAS Number: 102625-70-7
Other descriptive name: PANTOPRAZOLE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Ospedale San RaffaeleAuthorisedFemale: no
Male: yes
3Phase 2Italy
80NCT04136028September 25, 20154 November 2019IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous DiseaseA Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: KineretFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNot recruitingN/A18 YearsAll13Early Phase 1Russian Federation
No.TrialIDDate_
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81NCT01884311August 20, 201516 December 2017Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency DiseasesA Phase III, Multicenter, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Subgam-VF in Primary Immunodeficiency DiseasesPrimary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyperimmunoglobulin M SyndromeBiological: SubgamBio Products LaboratoryNot recruiting2 Years75 YearsAll38Phase 3United States
82NCT02369978August 201516 December 2017CHICAMOCHA 3 - Equivalence of Usual Interventions for Trypanosomiasis (EQUITY)Cardiovascular Health Investigation and Collaboration From Countries of America to Assess the Markers and Outcomes of Chagas Disease (CHICAMOCHA-3) - EQUITY (Equivalence of Usual Interventions for Trypanosomiasis)Chagas DiseaseDrug: Nifurtimox;Drug: Benznidazole;Drug: PlaceboUniversidad Autónoma de BucaramangaFundación Cardioinfantil Instituto de Cardiología;Instituto Nacional de Salud (Colombia);Instituto Nacional de Parasitologia Dr. Mario Fatala ChabenRecruiting20 Years55 YearsAll500Phase 2/Phase 3Colombia
83EUCTR2014-003746-27-DE15/04/201530 April 2018A randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using an infusion pump or syringeA randomised, cross-over study to compare quality of life and satisfaction in primary immunodeficient patients treated with subcutaneous injections of Gammanorm® 165 mg/mL administered with two different delivery devices: injections using pump or rapid push - Gammanorm in immunodeficient patients: quality of life using infusion by pump or rapid pushimmunodeficiency syndrome
MedDRA version: 19.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 ;Therapeutic area: Body processes [G] - Immune system processes [G12]
Trade Name: Gammanorm 165 mg/mLOctapharma Pharmazeutika Produktionsges.m.b.H, Oberlaaer Strasse 235, A-1100 Vienna, AustriaNot RecruitingFemale: yes
Male: yes
40Phase 3;Phase 4Australia;Germany;Italy;United Kingdom
84NCT02349906April 20154 November 2019Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant DiseasesClinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant DiseasesPrimary Immunodeficiencies;Inborn Errors of Metabolism;Haemoglobinopathies;Bone Marrow Failure SyndromesDrug: Treosulfan;Drug: Busilvexmedac GmbHCelerion;Venn Life Sciences;Syneos HealthRecruitingN/A17 YearsAll100Phase 2Austria;Czechia;Germany;Italy;Poland;Czech Republic
85NCT02490956April 20153 August 2015Diagnostic Immunization With Rabies Vaccine in Patients With PIDDiagnostic Immunization With Rabies Vaccine in Patients With Primary Immunodeficiency DisordersPrimary ImmunodeficiencyBiological: Verorab® (PVRV; Purified Vero Cell Vaccine)Chulalongkorn UniversityRecruiting12 Months60 YearsBoth40Phase 4Thailand
No.TrialIDDate_
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86NCT03198195March 10, 201516 December 2017Post-transplant Cyclophosphamide in Wiskott-Aldrich SyndromePost-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeProcedure: cyclophosphamideCapital Research Institute of PediatricsRecruiting5 Months10 YearsAll5N/A
87NCT02571062March 201519 October 2015Bioequivalence Study - Reference Clinical Fexinidazole Tablet Versus Proposed Market FormulationA Bioequivalence Study of the Reference Clinical Fexinidazole Tablet vs Proposed Market Formulation in Healthy Male Volunteers of African Sub-Saharan Origin:an Open-label,Randomized,Two-treatment,Single Dose,Replicate Design,Fed ConditionTrypanosomiasis, AfricanDrug: FexinidazoleDrugs for Neglected DiseasesNot recruiting18 Years45 YearsMale30Phase 1France
88NCT02972281March 201515 July 2019Systematic Search for Primary Immunodeficiency in Adults With InfectionsSystematic Search for Primary Immunodeficiency in Adults With Unexplained Recurrent and/or Severe Infections With Encapsulated BacteriaComplement Deficiency;Antibody Deficiency;Chronic Sinus Infection;Meningitis, Bacterial;Pneumonia, Bacterial;Otitis Media;Streptococcal Infection;Neisseria Infections;Haemophilus Influenza;Pneumococcal InfectionsBiological: Immunological diagnosis testsUniversity Hospital, LilleImagine Institute;Octapharma;CSL Behring;Laboratoire français de Fractionnement et de Biotechnologies;Air Liquide SA;The Binding Site LtdRecruiting18 Years65 YearsAll240N/AFrance
89NCT02909244February 201516 December 2017Study of Gut Microbiota in Primary Immune Deficiency, Possibly Associated With Inflammatory Bowel DiseaseStudy of Gut Microbiota in Primary Immune Deficiencies, Possibly Associated With Inflammatory Bowel Disease (Chronic Granulomatous Disease, XIAP Gene Deficiency, or TTC7A Gene Deficiency)Primary Immune DeficienciesBiological: Biological samplingImagine InstituteSaint Antoine University Hospital;Assistance Publique - Hôpitaux de ParisNot recruitingN/AN/AAll51N/AFrance
90NCT02162420January 20157 October 2019Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaHematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaRecruitingN/A70 YearsAll50N/AUnited States
No.TrialIDDate_
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91NCT02244450December 201420 August 2018Generalized Neonatal Screening of Severe Combined ImmunodeficienciesEvaluation of the Clinical Utility and Cost Effectiveness Ratio of Generalized Neonatal Screening for Severe Combined Immunodeficiencies (SCID) by Quantification of TRECs on Guthrie CardsSevere Combined Immunodeficiency, AtypicalBiological: SCID screeningNantes University HospitalNot recruitingN/A18 MonthsAll190539N/AFrance
92EUCTR2013-005508-33-PL17/11/201428 February 2019Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseasMale and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes.
MedDRA version: 20.0 Level: HLT Classification code 10021606 Term: Inborn errors of metabolism NEC System Organ Class: 100000004850
MedDRA version: 20.0 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870
MedDRA version: 20.0 Level: HLT Classification code 10018903 Term: Haemoglobinopathies congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Ovastat 1000 (Treosulfan injection)
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: TREOSULFAN
CAS Number: 299-75-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Trade Name: Ovastat 5000 (Treosulfan injection)
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: TREOSULFAN
CAS Number: 299-75-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5000-
Trade Name: Busilvex
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: BUSULFAN
CAS Number: 55-98-1
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 6-
medac GmbHAuthorised Female: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
93NCT02282904November 4, 20141 July 2019Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting2 Years65 YearsAll12Phase 1/Phase 2United States
94NCT03201380November 1, 201416 December 2017Evaluation of the Practice of the TEP Choline at Patients With Prostate CancerEvaluation of the Practice of the TEP Choline in Provence - Alps and Côte d'Azur at Patients With Prostate Cancer - Multicentre Retrospective Study.Prostate CancerOther: TEP scanner with cholineInstitut Paoli-CalmettesNot recruiting18 YearsN/AMale300N/A
95EUCTR2013-005508-33-DE29/09/201420 August 2018Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseasesClinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseasMale and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes.
MedDRA version: 20.0 Level: HLT Classification code 10021606 Term: Inborn errors of metabolism NEC System Organ Class: 100000004850
MedDRA version: 20.0 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870
MedDRA version: 20.0 Level: HLT Classification code 10018903 Term: Haemoglobinopathies congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Ovastat 1000 (Treosulfan injection)
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: TREOSULFAN
CAS Number: 299-75-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Trade Name: Ovastat 5000 (Treosulfan injection)
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: TREOSULFAN
CAS Number: 299-75-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5000-
Trade Name: Busilvex
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: BUSULFAN
CAS Number: 55-98-1
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 6-
medac Gesellschaft für klinische Spezialpräparate mbHAuthorisedFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
No.TrialIDDate_
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96NCT02231879September 3, 20143 June 2019Plerixafor Versus G-CSF in the Treatment of People With WHIM SyndromeA Phase III Double-Blind Randomized Crossover Study of Plerixafor Versus G-CSF in the Treatment of Patients With WHIM Syndrome.Myelokathexis;Infections;Neutropenia;Warts;HypogammaglobulinemiaDrug: Plerixafor;Drug: G-CSFNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting10 Years75 YearsAll19Phase 2/Phase 3United States
97NCT02179359September 2, 20147 October 2019Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesMT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent DisordersSickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic DisordersDrug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative RegimenMasonic Cancer Center, University of MinnesotaRecruitingN/A55 YearsAll25N/AUnited States
98NCT02199496July 23, 20149 September 2019Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable ImmunodeficiencyAn Open-Label Phase I/II Pilot Study to Assess the Safety/Tolerability and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable ImmunodeficiencyGastrointestinal Inflammation Associated With CVID;CVID EnteropathyBiological: Stelara (ustekinumab)National Institute of Allergy and Infectious Diseases (NIAID)Not recruiting18 Years75 YearsAll5Phase 1/Phase 2United States
99NCT02177760July 201414 December 2015Sirolimus Prophylaxis for aGVHD in TME SCIDSirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT)Severe Combined Immunodeficiency;Transplacental Maternal Engraftment;Stem Cell TransplantDrug: SirolimusUniversity of California, San FranciscoNot recruitingN/A1 YearBoth0Phase 2United States
100NCT02498782July 20143 August 2015Study to Evaluate Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chagas DiseasePhase 2, Randomized, Multicenter, Placebo-controlled, Safety and Efficacy Study to Evaluate Six Oral Fexinidazole Dosing Regimens for the Treatment of Adult Patients With Chronic Indeterminate Chagas Disease.Chagas Disease;Trypanosomiasis, South American;South American Trypanosomiasis;Disease, ChagasDrug: Fexinidazole;Drug: PlaceboDrugs for Neglected DiseasesRecruiting18 Years50 YearsBoth140Phase 2Bolivia
No.TrialIDDate_
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PhaseCountries
101EUCTR2014-000274-20-GB14/05/201431 January 2017Long-term follow-up of the WAS gene therapy studyLONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08) - Long-term follow-up of the WAS gene therapy study, version 2.0Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages.
MedDRA version: 19.1 Level: PT Classification code 10047992 Term: Wiskott-Aldrich syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Pharmaceutical Form: Solution for injection
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Concentration type: not less then
Concentration number: 0.5E06 cells /Kg-
GenethonAuthorisedFemale: no
Male: yes
10Phase 2United Kingdom
102EUCTR2013-003257-20-GB02/05/201428 February 2019TREOSULFAN LEVELS IN CHILDREN UNDERGOING STEM CELL TRANSPLANTATIONEvaluation of Treosulfan pharmacokinetics (PK) in children undergoing allogeneic haematopoietic stem cell transplantation (HSCT) - Treosulfan PK in childrenAny paediatric disease with an indication to an allogeneic stem cell transplantation (inclusing leukaemia, primary immunodeficiencies, metabolic disorders and autoimmune or genetic inflammatory bowel disorders).;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Trade Name: Treosulfan
Product Name: TREOSULFAN
Pharmaceutical Form: Injection
INN or Proposed INN: Treosulfan
CAS Number: 299-75-2
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUSTNot Recruiting Female: yes
Male: yes
90Phase 4United Kingdom
103NCT02169557May 201417 October 2016Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal StudyEfficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal StudyHuman African Trypanosomiasis (HAT)Drug: FexinidazoleDrugs for Neglected DiseasesNot recruiting15 YearsN/ABoth230Phase 2/Phase 3Congo, The Democratic Republic of the
104NCT02184689May 201417 October 2016Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal StudyEfficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal StudyHuman African Trypanosomiasis (HAT)Drug: fexinidazoleDrugs for Neglected DiseasesNot recruiting6 Years14 YearsBoth125Phase 2/Phase 3Congo, The Democratic Republic of the
105NCT02065869April 20144 November 2019Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological DisordersAcute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNot recruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;United Kingdom;Germany;Spain;United States
No.TrialIDDate_
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106NCT02180763April 201410 September 2018Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or PumpsA Randomized, Cross-over Study to Compare Quality of Life and Satisfaction in Primary Immunodeficient Patients Treated With Subcutaneous Injections of Gammanorm® 165 mg/mL According to the Delivery Device: Injections Using Pump or Rapid Push.Primary Immunodeficiency (PID)Biological: GammanormOctapharmaNot recruiting18 YearsN/AAll31Phase 4France
107NCT02510404April 201425 February 2019Multivirus-specific Cytotoxic T Lymphocytes (mCTL)Treatment of EBV, CMV, and Adenovirus Infections in Primary Immunodeficiency Disorders With Viral-specific Cytotoxic T-LymphocytesRefractory Viral InfectionsBiological: mCTLsCatherine BollardChildren's Research InstituteNot recruitingN/A45 YearsAll1Phase 1United States
108JPRN-UMIN0000131022014/03/102 April 2019Efficacy of Zoledronate for RAS associated ALPS like disease (RALD)RAS associated ALPS like disease (RALD)intravenous drip infusion of ZoledronateDepartment of Pediatrics, Tokyo Medical and Dental UniversityNot RecruitingNot applicableNot applicableMale and Female5Not selectedJapan
109NCT01888484March 20149 September 2019Study of Octanorm Subcutaneous IG in Patients With PIDClinical Phase III Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm 16.5%) In Patients With Primary Immunodeficiency DiseasesPrimary Immune Deficiency DisorderBiological: octanorm 16.5%OctapharmaNot recruiting2 Years75 YearsAll64Phase 3United States;Canada;Czechia;Hungary;Poland;Russian Federation;Slovakia;Czech Republic
110NCT01962415February 4, 20144 November 2019Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTA Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults =40 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell TransplantationPrimary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory ConditionsDrug: Hydroxyurea;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Melphalan;Drug: ThiotepaPaul SzabolcsRecruiting2 Months40 YearsAll100Phase 2United States
No.TrialIDDate_
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PhaseCountries
111NCT01814800February 201419 October 2017Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)Primary Immune Deficiency DisorderBiological: RI-002ADMA Biologics, Inc.Not recruiting2 Years75 YearsAll59Phase 3United States
112NCT01963143February 201416 December 2017Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency DiseasesA Phase III, Multicenter, Open-label, Randomized, Two-Period, Crossover Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency DiseasesPrimary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinaemia;Hyper-IgM SyndromeBiological: Gammaplex (5%);Biological: Gammaplex 10Bio Products LaboratoryNot recruiting2 Years55 YearsAll48Phase 3United States;Hungary;United Kingdom
113NCT01917708January 201419 November 2018BMT Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell DiseaseDrug: AbataceptEmory UniversityNot recruitingN/A21 YearsAll10Phase 1United States
114EUCTR2014-003409-13-Outside-EU/EEA12 January 2015Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects with Primary Immunodeficiency (Japan Study)A Multicenter Study of Long-Term Clinical Outcomes of Immune Globulin Subcutaneous (Human) (SCIG) IgPro20 in Subjects with Primary ImmunodeficiencyPrimary Immune Deficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Trade Name: Hizentra®
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human Normal Immunoglobulin
Other descriptive name: Human Normal Immunoglobulin
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 20-
CSL BehringNot AvailableFemale: yes
Male: yes
22Japan
115NCT01420627December 20139 September 2019EZN-2279 in Patients With ADA-SCIDA Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined ImmunodeficiencyADA-SCID;Adenosine Deaminase Deficiency;Severe Combined ImmunodeficiencyBiological: EZN-2279;Biological: AdagenLeadiant Biosciences, Inc.Not recruitingN/AN/AAll6Phase 3United States
No.TrialIDDate_
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Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
116NCT01998633December 201311 June 2018Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)Reduced-Intensity Conditioning for Children and Adults With Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (RICHI) (BMT CTN #1204)Hemophagocytic Lymphohistiocytosis;Chronic Active Epstein-Barr Virus Infection;Chronic Granulomatous Disease;HIGM-1;Leukocyte Adhesion Deficiency;IPEXBiological: Hematopoietic Stem Cell TransplantMedical College of WisconsinNational Heart, Lung, and Blood Institute (NHLBI);Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor ProgramNot recruiting4 Months45 YearsAll47Phase 2United States;Canada
117EUCTR2013-000961-36-DE15/11/20133 April 2017See the full titleTolerability and safety evaluation of the administration of Ig VENA at high infusion rates. Open label phase III study. - Ig VENA Infusion SpeedPatients affected by primary or secondary immunodeficiency (ID) or patients affected by Primary Immune Thrombocytopenia (ITP)
MedDRA version: 16.1 Level: PT Classification code 10054979 Term: Secondary immunodeficiency System Organ Class: 10021428 - Immune system disorders
MedDRA version: 16.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.1 Level: PT Classification code 10021245 Term: Idiopathic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Ig VENA 50 g/l solution for infusion 100 ml vial + infusion setKedrion SpANot RecruitingFemale: yes
Male: yes
35Phase 3Germany;Italy
118NCT02054832November 20136 October 2015Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked CornstarchA Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked CornstarchGlycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0Dietary Supplement: GlycosadeJohn MitchellNot recruiting2 Years50 YearsBoth11N/ACanada
119EUCTR2013-000883-27-NO16/08/201326 August 2013The effects of the local acting antibiotic Rifaximin on markers of inflammation in the blood by changing the bacterial composition in the gut, in patients with common variable immunodeficiency.EFFECTS OF RIFAXIMIN, BY MODULATION OF THE GUT MICROBIOTA, ON MARKERS OF SYSTEMIC INFLAMMATION IN PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY - AN EXPLORATORY OPEN-LABEL RANDOMIZED CONTROLLED TRIALAdult patients with the diagnosis of Common variable deficiency and fulfill the inclusion and exclusion criteria, will be invited to participate in the study.;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Trade Name: Xifaxan
Pharmaceutical Form: Coated tablet
Oslo University hospitalAuthorisedFemale: yes
Male: yes
Norway
120EUCTR2013-000620-34-IT05/08/201312 May 2014A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromesA Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes - NAPrimary Immunofediciency (PID) syndromes
MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Human Normal Immunoglobulin for subcutaneous administration
Product Code: LFB-IgSC
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human Normal immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 250-
LFB BiotechnologiesNot RecruitingFemale: yes
Male: yes
55Phase 3France;Hungary;Poland;Ukraine;Germany;United Kingdom;Italy
No.TrialIDDate_
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121EUCTR2012-005727-32-NL25/07/201310 July 2015The pharmacokinetics (the behaviour in the body) and safety of the intravenous immunoglobulin product Nanogam 100 mg/mlPharmacokinetics and safety of the intravenous human immunoglobulin product Nanogam 100 mg/ml - PK and safety of Nanogam 100 mg/mlPrimary a- or hypogammaglobulinemia
MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 100000004870
MedDRA version: 14.1 Level: LLT Classification code 10010509 Term: Congenital hypogammaglobulinemia System Organ Class: 100000004850
MedDRA version: 14.1 Level: LLT Classification code 10071133 Term: Congenital agammaglobulinemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Nanogam 100 mg/ml
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Other descriptive name: IVIG
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Trade Name: Nanogam® 50 mg/ml
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Other descriptive name: IVIG
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Sanquin Blood Supply FoundationNot RecruitingFemale: yes
Male: yes
Netherlands
122EUCTR2011-004154-25-IT25/06/201320 June 2016Clinical study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiencyA prospective, open-label, phase I/II study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenital fibrinogen deficiencyPatients with congenital afibrinogenemia or severe congenital hypofibrinogenemia.
MedDRA version: 14.1 Level: PT Classification code 10016075 Term: Factor I deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Fibrinogen Concentrate from Human Plasma
Product Code: BT524
Pharmaceutical Form: Powder for solution for injection/infusion
INN or Proposed INN: Human Fibrinogen Concentrate
CAS Number: 9001-32-5
Current Sponsor code: BT524
Other descriptive name: Human Fibrinogen Concentrate
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 14-26
Biotest AGNot RecruitingFemale: yes
Male: yes
20Phase 1;Phase 2Egypt;Lebanon;Germany;Italy
123EUCTR2012-005180-27-GB24/06/20133 July 2017Roxadustat in the Treatment of Anemia in Chronic Kidney Disease PatientsA Phase 3, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of Roxadustat for the Treatment of Anemia in Chronic Kidney Disease Patients not on Dialysis - AlpsAnemia in CKD patients not on dialysis
MedDRA version: 20.0 Level: LLT Classification code 10002272 Term: Anemia System Organ Class: 100000004851 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: roxadustat
Product Code: FG-4592/ASP1517 - 20 mg
Pharmaceutical Form: Tablet
INN or Proposed INN: roxadustat
CAS Number: 808118-40-3
Current Sponsor code: FG-4592
Other descriptive name: ASP1517
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Product Name: roxadustat
Product Code: FG-4592/ASP1517 - 50 mg
Pharmaceutical Form: Tablet
INN or Proposed INN: roxadustat
CAS Number: 808118-40-3
Current Sponsor code: FG-4592
Other descriptive name: ASP1517
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Product Name: roxadustat
Product Code: FG-4592/ASP1517 - 100 mg
Pharmaceutical Form: Tablet
INN or Proposed INN: roxadustat
CAS Number: 808118-40-3
Current Sponsor code: FG-4592
Other descriptive name: ASP1517
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Astellas Pharma Europe B.V.AuthorisedFemale: yes
Male: yes
600Phase 3Serbia;Panama;Belarus;Estonia;Greece;Spain;Guatemala;Ukraine;Turkey;Russian Federation;Colombia;Italy;United Kingdom;Hungary;Poland;Belgium;Romania;Dominican Republic;Peru;South Africa;Bulgaria;Georgia
124NCT01852370June 20, 201318 March 2019Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency DiseasesBilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric DonorsSevere Combined Immunodeficiency (SCID);Immunodeficiency With Predominant T-cell Defect, Unspecified;Severe Chronic Neutropenia;Chronic Granulomatous Disease (CGD);Hyper IgE Syndromes;Hyper IgM Deficiencies;Wiskott-Aldrich Syndrome;Mendelian Susceptibility to Mycobacterial Disease;Common Variable Immune Deficiency (CVID)Biological: CD3/CD19 negative allogeneic hematopoietic stem cellsPaul SzabolcsRecruiting5 Years45 YearsAll16Phase 1/Phase 2United States
125EUCTR2012-001725-26-DE12/03/20139 September 2013A Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vectorA Phase I/II Gene Therapy trial for X-CGD with a SIN gamma retroviral vector - gene therapy for X-CGDChronic granulomatous disease (CGD) is a congenital immunodeficiency, in which neutrophil granulocytes and monocytes are not capable of producing reactive oxygen species and therefore are unable to kill phagocytized bacteria or fungi.
MedDRA version: 14.1 Level: PT Classification code 10008906 Term: Chronic granulomatous disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: genetically modified autologous blood stem cells
Product Code: somatic gene-therapy by X-CGD
Pharmaceutical Form: Suspension for infusion
INN or Proposed INN: G1XCG
Other descriptive name: Genetically modified autologous blood stem cells
Concentration unit: Other
Concentration type: range
Concentration number: 100-500
Johann Wolfgang Goethe-UniversityAuthorisedFemale: yes
Male: yes
5Phase 1/2Germany
No.TrialIDDate_
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126NCT01821781March 20138 April 2019Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineRecruitingN/A21 YearsAll20Phase 2United States
127NCT01966367March 201325 March 2019CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationCD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant DiseaseBone Marrow Failure Syndrome;Severe Aplastic Anemia;Severe Congenital Neutropenia;Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Schwachman Diamond Syndrome;Primary Immunodeficiency Syndromes;Acquired Immunodeficiency Syndromes;Histiocytic Syndrome;Familial Hemophagocytic Lymphocytosis;Lymphohistiocytosis;Macrophage Activation Syndrome;Langerhans Cell Histiocytosis (LCH);Hemoglobinopathies;Sickle Cell Disease;Sickle Cell-beta-thalassemiaBiological: CD34 Stem Cell Selection TherapyDiane George, MDRecruitingN/A40 YearsAll25Early Phase 1United States
128EUCTR2012-000242-35-GB10/01/201314 November 2016Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous DiseaseA phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients with X-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patientsX-linked Chronic Granulomatous Disease
MedDRA version: 19.0 Level: PT Classification code 10008906 Term: Chronic granulomatous disease System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector
Product Code: G1XCGD transduced CD34+ cells
Pharmaceutical Form: Dispersion for infusion
Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR
Concentration unit: Other
Concentration number: >4x10e6 CD34+viab-cells/kg
GenethonAuthorisedFemale: no
Male: yes
11Phase 1;Phase 2France;Germany;Switzerland;United Kingdom
129NCT01218438January 201316 December 2017Phase 2/3 Study of IGSC, 20% in PIDDA Clinical Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) for the Evaluation of Efficacy, Safety, Tolerability and Pharmacokinetics in Subjects With Primary Immunodeficiency Diseases (PIDD)Primary Immunodeficiency Diseases (PID)Biological: Immune Globulin Intravenous (Human), 10% Solution;Drug: Immune Globulin Subcutaneous (Human), 20% SolutionBaxalta now part of ShireNot recruiting2 YearsN/AAll86Phase 2/Phase 3United States;Canada
130JPRN-UMIN0000093702012/12/072 April 2019Thalidomide therapy clinical trial for chronic granulomatous disease-associated intractable granulomatous lesionChronic granulomatous diseaseOral administration of thalidomideNational Center for Child Heath and DevelopmentNot Recruiting3years-oldNot applicableMale and Female5Not selectedJapan
No.TrialIDDate_
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131NCT01581593November 201219 February 2015Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)Multicenter, Open-label, Historically Controlled, Phase III Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Kedrion IVIG 10% in Adult and Pediatric Subjects With Primary Immunodeficiency (PID).Primary Immunodeficiency;Agammaglobulinemia;Hypogammaglobulinemia;Antibody DeficiencyBiological: Kedrion IVIG 10%Kedrion S.p.A.Not recruiting2 Years70 YearsBoth50Phase 3United States;Canada
132NCT01685827October 201226 February 2018Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2Efficacy and Safety of Fexinidazole Compared to Nifurtimox-Eflornithine Combination Therapy (NECT) in Patients With Late-stage Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: Pivotal, Non-inferiority, Multicentre, Randomised, Open-label StudyHuman African Trypanosomiasis (HAT);Sleeping SicknessDrug: Fexinidazole;Drug: Nifurtimox;Drug: EflornithineDrugs for Neglected DiseasesNot recruiting15 YearsN/AAll394Phase 2/Phase 3Central African Republic;Congo, The Democratic Republic of the;Congo
133NCT01652092September 4, 201228 January 2019Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesAllogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesSCID;Omenn's Syndrome;Reticular Dysgenesis;Wiskott-Aldrich Syndrome;Bare Lymphocyte Syndrome;Common Variable Immunodeficiency;Chronic Granulomatous Disease;CD40 Ligand Deficiency;Hyper IgM Syndrome;X-linked Lymphoproliferative Disease;Hemophagocytic Lymphohistiocytosis;Griscelli Syndrome;Chediak-Higashi Syndrome;Langerhan's Cell HistiocytosisDrug: Fludarabine phosphate 30 mg;Drug: MESNA;Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mgMasonic Cancer Center, University of MinnesotaRecruitingN/A50 YearsAll30N/AUnited States
134NCT01659606July 201215 April 2019Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaRadiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere DiseaseDyskeratosis Congenita;Hoyeraal Hreidarsson Syndrome;Revesz Syndrome;Aplastic AnemiaBiological: alemtuzumab;Drug: Fludarabine;Drug: Cyclosporins;Drug: Mycophenolate mofetilBoston Children’s HospitalDana-Farber Cancer Institute;Children's Hospital Medical Center, Cincinnati;Children's Hospital Los Angeles;Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium;Baylor College of Medicine;Children's Hospital of Philadelphia;Memorial Sloan Kettering Cancer Center;University of Wisconsin, Madison;Karolinska University HospitalRecruitingN/A65 YearsAll40Phase 2United States;Sweden
135NCT02327351July 20128 August 2016TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PIDPhase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency DiseasesPrimary Immune Deficiency Disorder;Hematopoietic Stem Cell TransplantationOther: Biological: TCR alfa beta T cell depletionFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyRecruiting1 Month18 YearsBoth60Phase 2/Phase 3Russian Federation
No.TrialIDDate_
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136EUCTR2012-000481-38-GB28/06/201224 June 2013Assessment of the safety of Immunoglobulin and recombinant human hylaluronidase in the treatment of patients with primary immunodeficiencyTolerability, Safety and Product Administration Evaluation of rHuPH20 Facilitated Subcutaneous Treatment with Immune Globulin (Human), 10% in Subjects with Primary Immunodeficiency Diseases – A Study in Europe - Tolerability and Safety of IG, 10% with rHuPH20 in PIDDPrimary Immunodeficiency Diseases
MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: KIOVIG 100 mg/ml solution for infusion
Product Name: Human normal immunoglobulin
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Trade Name: KIOVIG 100 mg/ml solution for infusion
Product Name: Human normal immunoglobulin
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Product Name: Recombinant Human Hyaluronidase (rHuPH20)
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HYALURONIDASE
CAS Number: 757971-58-7
Other descriptive name: 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20)
Concentration unit: U/ml unit(s)/millilitre
Concentration type: equal
Concentration number: 160-
Product Name: Human normal immunoglobulin
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Trade Name: KIOVIG 100 mg/ml solution for infusion
Product Name: Human normal immunoglobulin
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Product Name: Recombinant Human Hyaluronidase (rHuPH20)
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HYALURONIDASE
CAS Number: 757971-58-7
Other descriptive name: 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20)
Concentration unit: U/ml unit(s)/millilitre
Concentration type: equal
Concentration number: 160-
Baxter Innovations GmbHNot RecruitingFemale: yes
Male: yes
40Czech Republic;Belgium;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden
137EUCTR2012-000792-16-HU14/06/201227 May 2013Study to evaluate the efficacy, metabolism and safety of human immune globulin in patients with primary immunodeficiency diseases“CLINICAL STUDY TO EVALUATE THE PHARMACOKINETICS, SAFETY AND EFFICACY OF OCTAGAM 5% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES”primary immunodeficiency disease (PID)
MedDRA version: 14.1 Level: LLT Classification code 10049485 Term: Bruton's agammaglobulinemia System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 10021428 - Immune system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: OCTAGAM 50 mg/ml oldatos infúzió
Product Name: Octagam 5%
Pharmaceutical Form: Solution for infusion
CAS Number: 308067-58-5
Current Sponsor code: Octagam 5%
Other descriptive name: IMMUNOGLOBULIN G
Concentration unit: % percent
Concentration type: equal
Concentration number: 5-
OCTAPHARMA AGNot RecruitingFemale: yes
Male: yes
23Czech Republic;Hungary;Germany
138NCT03315078April 201210 December 2018Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyLentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyX-Linked Combined Immunodeficiency DiseasesBiological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)Recruiting2 Years40 YearsAll13Phase 1/Phase 2United States
139NCT01529827February 28, 201230 September 2019Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationPrimary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic LeukemiaDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNot recruiting3 Years75 YearsAll94Phase 2United States
140NCT01533961February 201228 September 2015Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the SCYX-7158Randomized, Double-blind, Placebo-controlled Sequential Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SCYX-7158 After Single Oral Ascending Doses in Healthy Male VolunteersTrypanosomiasis;Trypanosomiasis, African;Protozoan Infections;Parasitic DiseasesDrug: SCYX-7158;Drug: PlaceboDrugs for Neglected DiseasesNot recruiting18 Years45 YearsMale136Phase 1France
No.TrialIDDate_
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141NCT03333200January 11, 201211 November 2019Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghRecruitingN/AN/AAll1500Phase 1United States
142NCT03354533January 1, 20124 February 2019Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type IIStudy of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type IILeukocyte Adhesion Deficiency, Type IIDrug: L-fucoseOrpha LabsNot recruitingN/A18 YearsAll4Phase 1/Phase 2
143NCT01485796December 1, 201116 December 2017Tolerability and Safety of IGI, 10% With rHuPH20 in PIDDTolerability, Safety and Administration Mode Evaluation of Recombinant Human Hyaluronidase (rHuPH20) Facilitated Subcutaneous Treatment With Immune Globulin Infusion (Human), 10% in Subjects With Primary Immunodeficiency Diseases (PIDD)Primary Immunodeficiency DisordersBiological: Immune Globulin Infusion (Human), 10%;Biological: Recombinant human hyaluronidaseBaxalta now part of ShireNot recruiting2 YearsN/AAll54Phase 2/Phase 3United States
144NCT01465958November 201119 October 2017Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary ImmunodeficiencyAn Open-label, Single-sequence, Crossover Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Subcutaneous GAMUNEX®-C in Pediatric Subjects With Primary ImmunodeficiencyPrimary ImmunodeficiencyBiological: GAMUNEX-CGrifols Therapeutics Inc.Not recruiting2 Years16 YearsAll12Phase 4United States
145NCT01461018October 201119 February 2015Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects With Primary Immunodeficiency (Japan Study)A Multicenter Study of Long-Term Clinical Outcomes of Immune Globulin Subcutaneous (Human) (SCIG) IgPro20 in Subjects With Primary ImmunodeficiencyPrimary Immune DeficiencyBiological: Immune globulin subcutaneous (Human)CSL BehringNot recruitingN/A75 YearsBoth22Phase 3Japan
No.TrialIDDate_
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146NCT01220531September 8, 201129 July 2019Thymus Transplantation Safety-EfficacySafety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836Complete DiGeorge Anomaly;DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Thymus Tissue for Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Basiliximab;Drug: Mycophenolate mofetilM. Louise MarkertNational Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbHNot recruitingN/AN/AAllPhase 3United States
147NCT01406470September 201119 February 2015Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary ImmunodeficiencyAn Open-Label, Single-Arm, Historically Controlled, Prospective, Multicenter Phase III Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Immune Globulin Intravenous (Human) IVIG-SN™ in Subjects With Primary ImmunodeficiencyImmunologic Deficiency SyndromeDrug: Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™Green Cross CorporationAtlantic Research GroupNot recruiting2 Years70 YearsBoth45Phase 3United States;Canada
148NCT01483170September 201116 December 2017Multiple Dose Study to Evaluate Security, Tolerance and Pharmacokinetic of Fexinidazole (Drug Candidate for Human African Trypanosomiasis) Administered With a Loading Dose and With FoodDouble-blind, Placebo Controlled, Randomized Multiple Ascending Dose Study in Fed Conditions for Ten Days Dosing Regimen With a Loading Dose to Evaluate the Safety, the Tolerability and the Pharmacokinetics of Oral Fexinidazole in 36 Healthy Male Sub-Saharan Volunteers.Trypanosomiasis, AfricanDrug: Tablets Fexinidazole;Drug: PlaceboDrugs for Neglected DiseasesNot recruiting18 Years45 YearsMale30Phase 1France
149NCT01410825July 201120 May 2019Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromePilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeBiological: Retrovirus-mediated gene transferDavid WilliamsNot recruiting3 Months35 YearsMale5Phase 1/Phase 2United States
150NCT01412385June 20, 201113 May 2019Immune Globulin Subcutaenous (Human), 20%A Clinical Study of Immune Globulin Subcutaneous (Human) (IGSC), 20% for the Evaluation of Efficacy, Safety, and Pharmacokinetics in Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: Immune Globulin Subcutaneous (Human), 20%;Biological: Immune Globulin Intravenous (Human), 10%;Biological: Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin)Baxalta now part of ShireNot recruiting2 YearsN/AAll55Phase 2/Phase 3United Kingdom;Sweden;Hungary;Germany;Austria
No.TrialIDDate_
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151EUCTR2011-001118-32-DE25/05/201126 October 2015Twelve-month study on the immunogenicity (i.e. the ability of the study medication to provoke an immune response), safety and efficacy of the study medication Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropeniaTwelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropeniaSevere chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia)
MedDRA version: 14.1 Level: PT Classification code 10051645 Term: Idiopathic neutropenia System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 14.1 Level: LLT Classification code 10069819 Term: Congenital neutropenia System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: PT Classification code 10053176 Term: Cyclic neutropenia System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Trade Name: Filgrastim Hexal
Pharmaceutical Form: Solution for injection/infusion in pre-filled syringe
INN or Proposed INN: FILGRASTIM
CAS Number: 121181-53-1
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 300-
Trade Name: Filgrastim Hexal
Pharmaceutical Form: Solution for injection/infusion in pre-filled syringe
INN or Proposed INN: FILGRASTIM
CAS Number: 121181-53-1
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 480-
Product Name: EP2006 (Filgrastim)
Product Code: EP2006
Pharmaceutical Form: Solution for injection
INN or Proposed INN: FILGRASTIM
CAS Number: 121181-53-1
Current Sponsor code: EP2006
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 480-
Sandoz GmbHNot RecruitingFemale: yes
Male: yes
40Germany;Sweden
152NCT01313507May 201116 December 2017High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)Clinical Study to Evaluate the Safety and Tolerability of Immunoglobulin Intravenous (Human) 10% (NewGam) Administered at High Infusion Rates to Patients With Primary Immunodeficiency Diseases (Extension of Study NGAM-01)Primary Immunodeficiency DiseaseBiological: NewGamOctapharmaNot recruiting2 Years75 YearsAll21Phase 3United States
153NCT01458171April 201119 October 2017Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary ImmunodeficiencyPrimary Immune Deficiency DisorderBiological: Immune globulin subcutaneous (Human)CSL BehringNot recruitingN/A75 YearsAll23Phase 3Japan
154EUCTR2010-023483-41-HU01/03/201127 May 2013A multinational study with I10E (Human Immunoglobulin) to demonstrate the efficacy and the safety of the product in patients suffering from deficiency in their immune systemA MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) - I10E-0718a primary immunodeficiency as defined by the ESID and validated by a reference centre : • X-linked agammaglobulinemia (XLA) • Common variable immunodeficiency (CVID)
MedDRA version: 14.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency System Organ Class: 100000004870
MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 100000004870
MedDRA version: 14.1 Level: LLT Classification code 10001471 Term: Agammaglobulinemia System Organ Class: 100000004870 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR
Product Code: I10E
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
LFB BIOTECHNOLOGIESNot RecruitingFemale: yes
Male: yes
60Lithuania;Ukraine;Poland;Hungary;Czech Republic;France;Serbia
155NCT01289847March 201119 October 2017A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary ImmunodeficiencyA Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and AdolescentsPrimary Immune Deficiency Disorders;Common Variable Immunodeficiency;X-linked Agammaglobulinemia;Hyper-IgM Syndrome;Wiskott-Aldrich SyndromeBiological: GammaplexBio Products LaboratoryNot recruiting2 Years16 YearsAll25Phase 4United States;Chile;Israel
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156NCT01306019February 26, 201122 October 2019Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyX-linked Severe Combined Immunodeficiency;XSCID;SCID-X1;Gamma C-Deficient SCIDOther: Gene-modified CD34+ Hematopoietic stem cells;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)Recruiting2 Years40 YearsAll20Phase 1/Phase 2United States
157EUCTR2010-019459-23-DE18/01/201128 August 2014CLINICAL STUDY OF THE EFFICACY AND SAFETY OF SUBCUTANEOUS IMMUNE GLOBULIN, 20% IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESA CLINICAL STUDY OF IMMUNE GLOBULIN SUBCUTANEOUS (HUMAN) (IGSC), 20% FOR THE EVALUATION OF EFFICACY, SAFETY, AND PHARMACOKINETICS IN SUBJECTS WITH PRIMARY IMMUNODEFICIENCY DISEASES - EU Study of IGSC, 20% in PIDPrimary Immunodeficiency Diseases
MedDRA version: 16.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: KIOVIG
Product Name: KIOVIG
Product Code: IGIV, 10%
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
CAS Number: 0
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Trade Name: SUBCUVIA
Product Name: SUBCUVIA
Product Code: IGSC, 16%
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human normal immunoglobulin
CAS Number: 0
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: g/l gram(s)/litre
Concentration type: equal
Concentration number: 160-
Product Name: Immune Globulin Subcutaneos, 20%
Product Code: IGSC, 20%
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human normal immunoglobulin
CAS Number: 0
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Baxter Innovations GmbHNot RecruitingFemale: yes
Male: yes
47Sweden;United Kingdom;Germany;Netherlands;Austria;Belgium;Hungary
158NCT01338675January 201119 February 2015Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)Chronic Granulomatous DiseaseDrug: BusulfanSeoul National University HospitalRecruitingN/AN/ABoth5Phase 1/Phase 2Korea, Republic of
159NCT01222247October 201022 July 2019Antenatal Late Preterm Steroids (ALPS): A Randomized Placebo-Controlled TrialAntenatal Late Preterm Steroids (ALPS): A Randomized Placebo-Controlled TrialPregnancy;Respiratory Distress Syndrome;Pregnancy Outcomes;Preterm BirthDrug: Betamethasone;Drug: PlaceboThe George Washington University Biostatistics CenterNational Heart, Lung, and Blood Institute (NHLBI);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Not recruitingN/AN/AFemale2831Phase 3United States
160NCT01354587October 201019 February 2015Evaluation of Efficacy and Tolerability of Hizentra®Evaluation of Efficacy and Tolerability of Hizentra® in Subjects Transitioning From Vivaglobin® (16% SCIG Product) to Hizentra® (20% SCIG Product)Primary Immunodeficiency DisordersDrug: HizentraUniversity of South FloridaRecruiting1 Year75 YearsBoth50N/AUnited States
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161NCT01856582October 201011 June 2018CD34+ Stem Cell Infusion to Augment Graft FunctionPost Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure SyndromesWaning Donor Chimerism;Waning Immune Function;Primary Immunodeficiency Disease(s);Bone Marrow FailureBiological: CD34+Children's Hospital Medical Center, CincinnatiHoxworth Blood CenterNot recruitingN/A35 YearsAll23Phase 2United States
162JPRN-JapicCTI-10127106/9/201016 July 2019Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyPrimary ImmunodeficiencyIntervention name : Immune Globulin Subcutaneous (Human) IgPro20
INN of the intervention : Immune Globulin Subcutaneous (Human)
Dosage And administration of the intervention : The weekly dose of IgPro20 should be the weekly equivalent dose of the previous IVIG treatment.
CSL Behring K.K.Recruiting74BOTH15Phase 3
163NCT01199705September 201019 October 2017Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary ImmunodeficiencyPrimary Immune DeficiencyBiological: Immune Globulin Subcutaneous (Human) (SCIG)CSL BehringNot recruitingN/A75 YearsAll25Phase 3Japan
164NCT01319851September 201016 December 2017Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaNot recruitingN/A21 YearsAll3N/AUnited States
165EUCTR2010-019249-25-DE12/08/20108 May 2012An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID)An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infusion rates (Part B) in patients with primary immunodeficiency disease (PID)Primary immunodeficiency disease (PID)
MedDRA version: 14.1 Level: LLT Classification code 10010509 Term: Congenital hypogammaglobulinemia System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: Intratect
Product Code: BT090
Pharmaceutical Form: Solution for infusion
Current Sponsor code: BT090
Other descriptive name: Human normal immunoglobulin for intravenous use (IVIG)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Biotest AGNot RecruitingFemale: yes
Male: yes
30Germany;Hungary
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166NCT01182675August 201016 December 2017Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & FilgrastimHematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & FilgrastimSevere Combined ImmunodeficiencyDrug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and AlemtuzumabUniversity of California, San FranciscoNot recruitingN/A3 YearsAll7Phase 2United States
167NCT01175213July 201016 December 2017Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PIDLong-Term Tolerability and Safety of Immune Globulin Subcutaneous (IGSC) Solution Administered Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: IGSC - rHuPH20 then IGSC or IGIV;Biological: IGIV, 10% onlyBaxalta now part of ShireNot recruiting2 YearsN/AAll66Phase 3United States
168NCT01147042May 18, 201016 December 2017Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous DiseaseAssessment of the Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous DiseaseIFN-Gamma Therapy;CGD Gene Mutation;CGD Response to IFNg;CGD - Chronic Granulomatous Disease;Immunodeficiency DiseaseDrug: IFN-gammaNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/AAll2Phase 4United States
169NCT01129544April 201011 June 2019Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral VectorMulti-institutional Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene TransferSevere Combined ImmunodeficiencyBiological: Gene transferDavid WilliamsBoston Children’s Hospital;Children's Hospital Medical Center, Cincinnati;University of California, Los AngelesNot recruitingN/AN/AMale8Phase 1/Phase 2United States
170NCT03534479April 201011 June 2018Human IgGs and Endothelial Function in Vivo in HumansEffects of Intravenous Human Polyclonal Immunoglobulins G Infusion on Endothelial Function and Insulin Sensitivity in HumansCommon Variable ImmunodeficiencyDrug: Polyclonal IgGFederico II UniversityNot recruiting18 Years70 YearsAll24N/AItaly
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171EUCTR2009-017346-32-IT15/03/201019 March 2012A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WASA PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WASWiskott-Aldrich Sindrome
MedDRA version: 9.1 Level: PT Classification code 10061598
Product Name: autologous CD34+ cells transduced with a lentiviral vector encoding the WASP cDNA
Pharmaceutical Form: Suspension for injection
Other descriptive name: autologous CD34+ cells transduced with a lentiviral vector encoding the WASP cDNA
Concentration unit: % percent
Concentration type: equal
Concentration number: 100-
Trade Name: BUSILVEX
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Busulfan
Concentration unit: mOsm/kg milliosmol(s)/kilogram
Concentration type: equal
Concentration number: 1-
Trade Name: FLUDARABINA TEVA
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Fludarabine
Concentration unit: mg/m2 milligram(s)/square meter
Concentration type: equal
Concentration number: 30-
Trade Name: THYMOGLOBULINE
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Antithymocyte immunoglobulin (rabbit)
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 1-
Trade Name: MABTHERA
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Rituximab
Concentration unit: mg/m2 milligram(s)/square meter
Concentration type: equal
Concentration number: 375-
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABORAuthorisedFemale: no
Male: yes
Phase 1/2Italy
172EUCTR2009-011434-10-DE02/03/201024 April 2012CLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESCLINICAL STUDY TO EVALUATE THE EFFICACY, PHARMACOKINETICS AND SAFETY OF IMMUNOGLOBULIN INTRAVENOUS (HUMAN) 10% (NEWGAM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASESPrimary immunodeficiency diseases
MedDRA version: 12.0 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency
MedDRA version: 12.0 Level: LLT Classification code 10049485 Term: Bruton's agammaglobulinemia
Product Name: NewGam
Product Code: NewGam
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: immunoglobulin G
Other descriptive name: NewGam
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
OCTAPHARMA AGNot RecruitingFemale: yes
Male: yes
50Germany
173NCT01131858March 201019 February 2015Study of Vitamin D3 Substitution to Patients With Primary ImmunodeficiencyA Placebo Controlled Double Blinded Study of Vitamin D3 Substitution to Patients With Primary ImmunodeficiencyPrimary Immune Deficiency DisorderDrug: Vigantol;Drug: PlaceboKarolinska University HospitalNot recruiting18 Years75 YearsBoth140Phase 1/Phase 2Sweden
174EUCTR2007-000684-16-GB21/01/201026 June 2012Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1X-Linked severe combined Immunodeficiency (SCID-X1)
MedDRA version: 9.1 Level: LLT Classification code 10010099 Term: Combined immunodeficiency
Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector
Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector
Pharmaceutical Form: Solution for blood fraction modification
Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector transduced cells
Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector transduce
Pharmaceutical Form: Solution for infusion
Great Ormond Street Hospital NHS Trust / University College London - Institute of Child HealthAuthorisedFemale: no
Male: yes
10United Kingdom
175EUCTR2007-004308-11-GB11/01/201013 February 2017Gene therapy for WASPHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages.
MedDRA version: 19.1 Level: PT Classification code 10047992 Term: Wiskott-Aldrich syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Pharmaceutical Form: Solution for injection
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Concentration unit: Other
Concentration type: not less then
Concentration number: 0.5E06 cells /Kg-
Pharmaceutical Form: Solution for injection
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Concentration unit: Other
Concentration type: not less then
Concentration number: 0.5E06 cells /Kg-
Pharmaceutical Form: Solution for injection
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Concentration unit: Other
Concentration type: not less then
Concentration number: 0.5E06 cells /Kg-
GenethonAuthorisedFemale: no
Male: yes
Phase 1;Phase 2United Kingdom
No.TrialIDDate_
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176NCT01001598November 200919 February 2015Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaPhase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaFanconi Anemia;Dyskeratosis CongenitaDrug: danazolChildren's Hospital BostonNot recruiting3 YearsN/ABoth5Phase 1/Phase 2United States
177NCT00982904September 200916 December 2017Human African Trypanosomiasis: First in Man Clinical Trial of a New Medicinal Product, the FexinidazoleRandomized, Double-blind, Placebo-controlled Study of the Tolerability, and Pharmacokinetics of Fexinidazole After Single and Repeated Oral Ascending Doses, Completed by a Comparative Bioavailability Study of an Oral Suspension Versus a Tablet and an Exploratory Assessment of Food Effect, in Healthy Male VolunteersHuman African TrypanosomiasisDrug: Fexinidazole/PlaceboDrugs for Neglected DiseasesSanofiNot recruiting18 Years45 YearsMale108Phase 1France
178EUCTR2009-012036-32-FR30/07/200919 March 2012SAFETY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, WHEN ADMINISTERED TO PRIMARY IMMUNODEFICIENT PATIENTS, AT A PROGRESSIVELY INCREASED FLOW RATESAFETY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, WHEN ADMINISTERED TO PRIMARY IMMUNODEFICIENT PATIENTS, AT A PROGRESSIVELY INCREASED FLOW RATEPRIMARY IMMUNODEFICIENCY
MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome
Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE
Product Code: IGNG
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
LFB BIOTECHNOLOGIESAuthorisedFemale: yes
Male: yes
France
179NCT00909363June 20091 April 2019Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) PatientsEffects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.Wiskott-Aldrich Syndrome;Thrombocytopenia;BleedingDrug: Promacta;Diagnostic Test: blood drawing in patients with WAS;Diagnostic Test: blood drawing in healthy controlsWeill Medical College of Cornell UniversityNovartis PharmaceuticalsNot recruiting3 Months80 YearsMale24Phase 2United States
180NCT00906880April 200919 February 2015Clinical Study to Assess the Tolerability, Feasibility and Effectiveness of Nifurtimox and Eflornithine (NECT) for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic PhaseClinical Study to Assess the Clinical Tolerability, Feasibility and Effectiveness Under Field Conditions of the Combination of Nifurtimox and Eflornithine (NECT) for the Treatment of T.b.Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic StageHuman African TrypanosomiasisDrug: Nifurtimox-Eflronithine Combination Treatment (NECT)Drugs for Neglected DiseasesMinistry of Public Health, Democratic Republic of the Congo;Swiss Tropical & Public Health InstituteNot recruitingN/AN/ABoth630Phase 4Congo
No.TrialIDDate_
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181NCT00799071February 200919 February 2015Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD)Investigation of POsaconazole Prophylaxis in Children With Chronic Granulomatous Disease (CGD): Pharmacokinetics and Tolerability (iPOD)Chronic Granulomatous DiseaseDrug: posaconazole (PSZ)Radboud UniversityNot recruiting2 Years16 YearsBoth12Phase 2Netherlands;United Kingdom
182EUCTR2007-003235-23-FR09/01/200919 March 2012Essai randomisé multicentrique de phase II d'évaluation immunologique d'une stratégie vaccinale de type prime boost associant une administration du vaccin conjugué anti-pneumococcique à S0 suivie de l'injection du vaccin polysaccharidique à S4 comparé à l'administration du vaccin polysaccharidique seul à S4 chez des patients ayant un déficit immunitaire commun variable - Evaluation d'une stratégie vaccinale anti-pneumococcique chez des patients aynt un DICVEssai randomisé multicentrique de phase II d'évaluation immunologique d'une stratégie vaccinale de type prime boost associant une administration du vaccin conjugué anti-pneumococcique à S0 suivie de l'injection du vaccin polysaccharidique à S4 comparé à l'administration du vaccin polysaccharidique seul à S4 chez des patients ayant un déficit immunitaire commun variable - Evaluation d'une stratégie vaccinale anti-pneumococcique chez des patients aynt un DICVDéficit immunitaire commun variable
MedDRA version: 9.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency
Trade Name: PNEUMO 23
Pharmaceutical Form: Solution for injection
Trade Name: PREVENAR
Pharmaceutical Form: Suspension for injection
InsermAuthorisedFemale: yes
Male: yes
Phase 2France
183NCT00811174January 200916 December 2017Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency DiseasesClinical Study to Evaluate the Efficacy, Safety and Kinetics of Octagam 10% for Replacement Therapy in Primary Immunodeficiency DiseasesImmunologic Deficiency SyndromesDrug: Octagam 10%OctapharmaNot recruiting2 Years75 YearsAll5Phase 3Austria
184NCT01289171January 200919 February 2015Acquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric PatientsAcquired Epidermodysplasia Verruciformis (EV) Syndrome in HIV-infected Pediatric Patients: Prospective Treatment Trial With Topical Glycolic Acid and HPV Genotype CharacterizationFlat Warts (Diagnosis);HIV InfectionsDrug: Glycolic acidUniversity of PennsylvaniaNeoStrata Company, Inc.;Penn Center for AIDS Research (CFAR)Not recruiting7 YearsN/ABoth38N/ABotswana
185EUCTR2009-011152-22-FR2 October 2017Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WASPhase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WASPhase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the lentiviral vector w1.6_hWASP_WPRE (VSVg) in up to 5 patients with WAS.Product Name: Autologous CD34+ cells transduced with the Lentiviral vector containing the human Wiskott Aldrich Sy
Product Code: GTG003.08
Pharmaceutical Form: Solution for injection
GENETHONNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2France
No.TrialIDDate_
enrollement
Last_Refreshed_
on
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sponsor
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186NCT00814320December 200816 December 2017Gammagard Liquid and rHuPH20 in PIDEfficacy, Tolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human), 10% (GAMMAGARD LIQUID/KIOVIG) Administered Intravenously or Subcutaneously Following Administration of Recombinant Human Hyaluronidase (rHuPH20) in Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency Diseases (PID)Biological: Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)Baxalta now part of ShireNot recruiting2 YearsN/AAll89Phase 3United States;Canada
187EUCTR2008-004518-28-NL19/11/200814 August 2012Investigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPODInvestigation of posaconazole prophylaxis in children with chronic granulomatous disease (CGD): pharmacokinetics and tolerability (iPOD) - iPODChildren with chronic granulomatous disease (CGD).
MedDRA version: 9.1 Level: LLT Classification code 10008906 Term: Chronic granulomatous disease
Trade Name: Noxafil 40 mg/ml oral solution
Pharmaceutical Form: Oral suspension
Radboud University Nijmegen Medical CentreNot RecruitingFemale: yes
Male: yes
United Kingdom;Netherlands
188NCT00794508November 200819 October 2017MND-ADA Transduction of CD34+ Cells From Children With ADA-SCIDMND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With BusulfanSevere Combined ImmunodeficiencyBiological: ADA gene transferDonald B. Kohn, M.D.FDA Office of Orphan Products Development;National Institutes of Health (NIH)Not recruiting1 Month18 YearsAll10Phase 2United States
189EUCTR2007-002611-27-DE27/10/200811 February 2013Clinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NAClinical study to evaluate the efficacy, safety and kinetics of Octagam® 10% for replacement therapy in Primary Immunodeficiency Diseases (PID) - NAPrimary Immundeficiency Diseases (PID)
MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome
Trade Name: Octagam 10%
Product Name: Octagam 10%
Pharmaceutical Form: Solution for infusion
Current Sponsor code: GAM10
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 10-
Octapharma AGNot RecruitingFemale: yes
Male: yes
45France;Germany;United Kingdom
190NCT00774358October 200816 December 2017Interleukin-2 Treatment for Wiskott-Aldrich SyndromeReinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 TherapyWiskott-Aldrich Syndrome (WAS);X-linked ThrombocytopeniaDrug: Interleukin-2Soma JyonouchiTexas Children's HospitalNot recruiting24 MonthsN/AAll9Phase 1United States
No.TrialIDDate_
enrollement
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191EUCTR2008-000830-30-DE10/07/200828 August 2012A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency (IgPro20 EU Extension Study)A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary Immunodeficiency (IgPro20 EU Extension Study)PID (primary immuno deficiency)
MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome
Product Name: IgPro20
Product Code: IgPro20
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human Normal Immunoglobulin
Concentration unit: % percent
Concentration type: equal
Concentration number: 20-
CSL Behring AGNot RecruitingFemale: yes
Male: yes
36United Kingdom;Germany;France;Spain;Sweden
192EUCTR2008-003368-21-IT04/06/200819 March 2012Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndromePilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndromeFor patients with a stabilized clinical condition, not recovered in care units for acute problems.
MedDRA version: 9.1 Level: HLGT Classification code 10003816
MedDRA version: 9.1 Level: HLT Classification code 10027657
Trade Name: ORENCIA
Pharmaceutical Form: Powder for infusion*
INN or Proposed INN: ABATACEPT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
ISTITUTO PER L`INFANZIA BURLO GAROFOLONot RecruitingFemale: no
Male: yes
Italy
193NCT00719680June 200819 October 2017Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID)Primary Immune DeficiencyBiological: IgPro20CSL BehringNot recruiting2 Years75 YearsAll21Phase 3United States
194NCT00634569May 200819 October 2017Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric SubjectsEvaluation of the Efficacy and Safety of Flebogamma 5% DIF [Immune Globulin Intravenous (Human)] for Replacement Therapy in Pediatric Subjects With Primary Immunodeficiency Diseases.Primary Immune Deficiency DiseaseBiological: Flebogamma 5% DIFInstituto Grifols, S.A.Not recruiting2 Years16 YearsAll24Phase 4United States
195NCT00680446April 200819 February 2015Safety Study of Subcutaneous Ig NextGen 16% in Patients With Primary ImmunodeficiencyAn Open-Label Study of Ig NextGen 16% Administered by Subcutaneous Infusion in Patients With Primary Immunodeficiency (PID).Primary Immune DeficiencyDrug: Immunoglobulin G (Ig NextGen 16%)CSL LimitedNot recruiting3 YearsN/ABoth41Phase 3Australia;New Zealand
No.TrialIDDate_
enrollement
Last_Refreshed_
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196NCT00605657January 200819 October 2017Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)Pilot (Phase I-II) Study of Valproic Acid (Depakote) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS)ALPS;Hypersplenism;LymphadenopathyDrug: Valproic Acid;Procedure: CT Scan;Procedure: Blood SampleKoneti RaoNot recruiting2 Years70 YearsAll6Phase 1/Phase 2United States
197NCT00546871October 200716 December 2017Comparison of Intravenous and Subcutaneous Administration of IGIV, 10% in Primary Immunodeficiency (PID) SubjectsTolerability and Pharmacokinetic Comparison of Immune Globulin Intravenous (Human) 10% (IGIV, 10%) Administered Intravenously or Subcutaneously in Subjects With Primary Immunodeficiency DiseasesPrimary Immunodeficiency (PID)Drug: Immune Globulin Intravenous (Human), 10%Baxalta now part of ShireNot recruiting24 MonthsN/AAll49Phase 2/Phase 3United States
198NCT00579137October 200719 October 2017Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency DisordersCD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency DisordersSevere Combined Immunodeficiency Disease;Severe Primary Immunodeficiency Disorder;Undefined T Cell Deficiency Disorder;Wiskott-Aldrick SyndromeBiological: Campath -1H;Drug: Fludarabine;Biological: Anti-CD45;Procedure: Stem cell infusionBaylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of Medicine;Texas Children's HospitalNot recruitingN/AN/AAll3Phase 1/Phase 2United States
199EUCTR2006-006023-39-IT24/09/200719 March 2012Pharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - NDPharmacokinetics of Haemocomplettan P in subjects with congenital fibrinogen deficiency - NDPK study in patient with afibrinogemia
MedDRA version: 9.1 Level: LLT Classification code 10016075 Term: Factor I deficiency
Trade Name: Haemocomplettan P
Pharmaceutical Form: Powder and solvent for solution for infusion
Current Sponsor code: Factor I
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1-
CSL Behring GmbHNot RecruitingFemale: yes
Male: yes
Italy
200NCT00527878September 200719 October 2017Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) SyndromeA Double-Blind, Randomized, Placebo-Controlled Cross-Over Study Assessing the Role of Pathogen-Specific IgE and Histamine Release in the Hyper-IgE Syndrome and the Effect of Ranitidine on Laboratory and Clinical ManifestationsJOB's Syndrome;Hyper-IgE Recurrent Infection Syndrome;Immune DeficiencyDrug: Ranitidine;Drug: PlaceboNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting2 YearsN/AAll16Phase 2United States
No.TrialIDDate_
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201NCT00542997September 200719 October 2017Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement TherapyA Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary ImmunodeficiencyCommon Variable Immunodeficiency;X-linked Agammaglobulinemia;Autosomal Recessive AgammaglobulinemiaBiological: Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)CSL BehringNot recruiting2 Years65 YearsAll51Phase 3France;Germany;Italy;Poland;Romania;Spain;Sweden;Switzerland;United Kingdom
202NCT00455312August 200716 December 2017Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAAHematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: MethylprednisoloneMasonic Cancer Center, University of MinnesotaNot recruitingN/A70 YearsAll36Phase 2/Phase 3United States
203EUCTR2006-006745-13-DE11/07/200719 March 2012A Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyA Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects with Primary ImmunodeficiencyPID (Primary Immunodeficiency)
MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome
Product Name: IgPro20
Product Code: IgPro20
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human Immunglobulin G (IgG)
Concentration unit: % percent
Concentration type: equal
Concentration number: 20-
CSL Behring AGNot RecruitingFemale: yes
Male: yes
51United Kingdom;Germany;France;Spain;Italy;Poland;Sweden
204NCT00490100June 200719 October 2017Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency (XSCID): A Phase 2 Study Assessing Safety and Clinical Response to Treatment With Insulin-like Growth Factor-1 (IGF-1)Growth Failure;X-linked Severe Combined Immunodeficiency (XSCID);Growth Hormone ResistenceDrug: IncrelexNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting2 Years20 YearsAll6Phase 1/Phase 2United States
205EUCTR2007-001410-17-FR31/05/200719 March 2012LONG-TERM SAFETY AND EFFICACY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, ADMINISTERED IN CURRENT PRACTICE TO PRIMARY IMMUNODEFICIENT PATIENTSLONG-TERM SAFETY AND EFFICACY STUDY OF IGNG, A NEW LIQUID PREPARATION OF HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE, ADMINISTERED IN CURRENT PRACTICE TO PRIMARY IMMUNODEFICIENT PATIENTSPrimary Immunodeficiency
MedDRA version: 9.1 Level: LLT Classification code 10064859 Term: Primary immunodeficiency syndrome
Product Name: Human normal immunoglobulin for intravenous administration
Product Code: IGNG
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin for intravenous administration
Current Sponsor code: IGNG
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
LFB SAAuthorisedFemale: yes
Male: yes
France
No.TrialIDDate_
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206NCT00391131April 200719 February 2015Subcutaneous Ig NextGen 16% in PID PatientsA Multi-centre, Open-label Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Subcutaneous Infusions of Ig NextGen 16% in Patients With Primary Immunodeficiency (PID).Primary Immunodeficiency (PID)Drug: IgNextGen 16%CSL LimitedNot recruiting3 YearsN/ABoth35Phase 3Australia;New Zealand
207EUCTR2006-006522-25-DE01/02/200719 March 2012A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID)A multicenter study on the efficacy and safety of Vivaglobin® in Previously Untreated Patients (PUPs) with Primary Immunodeficiency (PID)Patients with PID diseases as Common Variable Immunodeficiency (CVID) or X-linked agammaglobulinemia ( XLA), age 1 to 70 years
MedDRA version: 8.1 Level: LLT Classification code 10010112 Term: Common variable immunodeficiency
Trade Name: Vivaglobin
Product Name: Vivaglobin
Pharmaceutical Form: Cutaneous solution
INN or Proposed INN: immune globulin subcutaneous (human)
Current Sponsor code: CE1200
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 160-
CSL Behring AGNot RecruitingFemale: yes
Male: yes
28United Kingdom;Germany;Belgium;Spain;Italy;Greece
208NCT00885833February 200719 February 2015Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeDrug: Fludarabine, Busulfan, ThymoglobulinThe Korean Society of Pediatric Hematology OncologyNot recruiting1 Year25 YearsBoth5Phase 1/Phase 2Korea, Republic of
209NCT02512679February 200716 December 2017Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood CellsProtocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological DiseasesStem Cell Transplantation;Bone Marrow Transplantation;Peripheral Blood Stem Cell Transplantation;Allogeneic Transplantation;Genetic Diseases;Thalassemia;Pediatrics;Diamond-Blackfan Anemia;Combined Immune Deficiency;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Lymphoproliferative Disease;Metabolic DiseasesDrug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4Children's Hospital Los AngelesLucile Packard Children's HospitalNot recruiting3 MonthsN/AAll20Phase 2
210NCT02127892January 2, 200716 December 2017SCID Bu/Flu/ATG Study With T Cell DepletionPhase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling DonorSevere Combined ImmunodeficiencyBiological: unrelated BM with T cell depletion;Biological: unrelated cord blood;Biological: haplo BM with T cell depletion;Device: unrelated PBSC with T cell depletionNeena Kapoor, M.D.Not recruitingN/A21 YearsAll9Phase 1/Phase 2United States
No.TrialIDDate_
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PhaseCountries
211NCT00778882January 200728 October 2019Gene Therapy for Chronic Granulomatous Disease in KoreaAn Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease PatientsChronic Granulomatous DiseaseDrug: VM106Helixmith Co., Ltd.Not recruitingN/AN/AMale2Phase 1/Phase 2Korea, Republic of
212NCT00782106December 4, 20065 February 2018Study to Determine the Dose of Recombinant Human Hyaluronidase Needed to Infuse a Full Dose of IGIV SubcutaneouslyPhase 1/2 Determination of the Dose of Recominant Human Hyaluronidase (rHuPH20) Required Enabling Up to 600 mg/kg Bodyweight of IGIV, 10% to be Administered Subcutaneously in a Single Infusion Site in Subjects With Primary Immunodeficiency (PID)Primary Immunodeficiency Diseases (PID)Biological: Recombinant human hyaluronidase + immune globulin intravenousBaxalta now part of ShireNot recruiting16 YearsN/AAll11Phase 1/Phase 2United States
213NCT00392951December 200619 November 2018Sirolimus for Autoimmune Disease of Blood CellsSirolimus for Patients With Chronic and/or Refractory Autoimmune Cytopenias: A Pilot SeriesAutoimmune Pancytopenia;Autoimmune Lymphoproliferative Syndrome (ALPS);Evans Syndrome;Idiopathic Thrombocytopenic Purpura;Anemia, Hemolytic, Autoimmune;Autoimmune Neutropenia;Lupus Erythematosus, Systemic;Inflammatory Bowel Disease;Rheumatoid ArthritisDrug: sirolimusChildren's Hospital of PhiladelphiaNot recruiting1 Year30 YearsAll30Phase 1/Phase 2United States
214NCT00389324November 200619 October 2017A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary ImmunodeficiencyAn Open-Label Single-Sequence, Crossover Trial to Evaluate the Pharmacokinetics and Safety of Subcutaneous Gamunex® 10% (Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified) in Subjects With Primary ImmunodeficiencyImmunologic Deficiency SyndromeBiological: Immune Globulin Intravenous (Human)Grifols Therapeutics Inc.Not recruiting13 Years75 YearsAll35Phase 2United States;Canada
215NCT00419341November 200619 October 2017Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement TherapyA Phase III Open-Label, Prospective, Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human), IgPro20 in Subjects With Primary Immunodeficiency (PID)Primary Immune DeficiencyBiological: Human Normal Immunoglobulin for Subcutaneous AdministrationCSL BehringNot recruiting2 Years75 YearsAll49Phase 3United States
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216NCT00394316October 30, 200616 July 2018Gene Therapy for Chronic Granulomatous DiseaseAutologous Transplantation of Genetically Modified Cells for the Treatment of X-Linked Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem CellsNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting3 Years55 YearsMale3Early Phase 1United States
217NCT00395538October 30, 20062 September 2019Effects of PTH Replacement on Bone in HypoparathyroidismEffects of PTH Replacement on Bone in HypoparathyroidismHypoparathyroidism;DiGeorge SyndromeDrug: PTH 1-34National Institute of Dental and Craniofacial Research (NIDCR)Not recruiting18 Years70 YearsAll46Phase 3United States;Austria;Italy
218NCT00358657May 24, 200628 October 2019Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited DisordersHLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Not recruitingN/A55 YearsAll14Phase 2United States
219NCT00325078May 200619 October 2017Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous DiseaseTumor Necrosis Factor Alpha Inhibitor (Lnfliximab, Adalimumab) Treatment for Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease: A Phase I/II Study Assessing Clinical and Immune Responses to Treatment and Genetic InfluencesChronic Granulomatous Disease;Crohn'S-like IBD;Inflammatory Bowel Disease (IBD)Drug: InfliximabNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting10 YearsN/AAll40Phase 1/Phase 2United States
220NCT00579527March 200629 July 2019Phase I/II Thymus Transplantation With Immunosuppression #950Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge SyndromeDrug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil;Drug: Tacrolimus;Drug: Cyclosporine;Drug: Rabbit anti-thymocyte globulin;Procedure: Blood Draw;Other: Thymus Tissue and Parental Parathyroid Transplantation;Biological: Thymus Tissue for TransplantationM. Louise MarkertNational Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbHNot recruitingN/AN/AAll28Phase 1/Phase 2United States
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221NCT00576836February 200620 May 2019Thymus Transplantation Dose in DiGeorge #932Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1DiGeorge Anomaly;DiGeorge Syndrome;Complete DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Thymus Tissue for Transplantation;Other: Parathyroid Tissue for TransplantationM. Louise MarkertNational Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbHNot recruitingN/AN/AAll28Phase 2United States
222NCT00278954January 200619 October 2017Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.A Phase III, Multicenter, Open-Label Study To Evaluate The Efficacy, Safety, and Pharmacokinetics of Gammaplex® in Primary Immunodeficiency DiseasesPrimary Immunodeficiency;Common Variable Hypogammaglobulinemia;X-linked Hypogammaglobulinemia;Hypogammaglobulinemia;Immunodeficiency With Hyper-IgM;Wiskott-Aldrich SyndromeBiological: Gammaplex (Intravenous immunoglobulin)Bio Products LaboratoryNot recruiting3 YearsN/AAll50Phase 3United States
223NCT00263237December 2, 200516 December 2017STA-5326 Meslylate to Treat Gut Inflammation Associated With Common Variable ImmunodeficiencyA Pilot Study of Safety and Efficacy of the Oral IL-12/23 Inhibitor, STA-5326 Mesylate, for Symptomatic Gastrointestinal Inflammation Associated With Common Variable ImmunodeficiencyCommon Variable ImmunodeficiencyDrug: STA-5326National Institute of Allergy and Infectious Diseases (NIAID)Not recruiting18 Years75 YearsAll10Phase 1United States
224NCT00294567December 200519 February 2015Azelnidipine Anti-Coronary Atherosclerotic Trial in Hypertensive Patients by Serial Volumetric IVUS Analysis(ALPS-J)Azelnidipine and Amlodipine Anti-Coronary Atherosclerotic Trial in Hypertensive Patients Undergoing Coronary Intervention by Serial Volumetric Intravascular Ultrasound Analysis in Junten Medical University (ALPS-J)Hypertension;Coronary AtherosclerosisDrug: Calcium channel blockers (amlodipine, azelnidipine)Juntendo University HospitalJapan Heart FoundationNot recruiting20 Years79 YearsBoth200Phase 4Japan
225NCT00260702November 24, 200516 December 2017Omalizumab to Treat Hyper-IgE (Job's) SyndromePilot Study of Omalizumab (Xolair) in Hyper IgE (Job's) SyndromeHyper-IgE Syndrome;Job's SyndromeDrug: Omalizumab (Xolair)National Institute of Allergy and Infectious Diseases (NIAID)Not recruiting6 Years76 YearsAll1Phase 1United States
No.TrialIDDate_
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226NCT00322556November 200519 October 2017Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID)Agammaglobulinemia;IgG Deficiency;Common Variable ImmunodeficiencyDrug: Immunoglobulins Intravenous (Human)CSL BehringNot recruiting4 Years71 YearsAll55Phase 3United States
227NCT00119431September 200519 February 2015Kinetics, Efficacy and Safety of C1-Esteraseremmer-NPharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) AngioedemaHereditary Angioedema Type I;Angioneurotic EdemaDrug: C1 inhibitor concentrateSanquinNot recruiting18 YearsN/ABoth12Phase 2Netherlands
228NCT00160355May 200516 December 2017Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich SyndromeHaploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot StudyWiskott-Aldrich SyndromeProcedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, ThiotepaSt. Jude Children's Research HospitalNot recruitingN/A18 YearsMale4Phase 1United States
229EUCTR2004-004465-15-DE05/04/200519 March 2012A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID)A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients with primary immunodeficiency disease (PID)primary immunodeficiency syndrome as congenital agammaglobulinaemia or hypogammaglobulinaemia, common variable immunodeficiency, severe combined immunodeficiencies, Wiskott Aldrich syndrome
MedDRA version: 7.0 Level: HLT Classification code 10036700
Product Name: Intratect
Product Code: BT681
Pharmaceutical Form: Solution for infusion
Current Sponsor code: BT681 (Intratect)
Other descriptive name: human normal immunoglobulin (IVIg)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Biotest AGNot RecruitingFemale: yes
Male: yes
50Hungary;Germany
230NCT00295971April 200519 February 2015Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency DiseaseStem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency DiseasesCongenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiationUniversity of California, San FranciscoNational Cancer Institute (NCI)Not recruiting1 Year17 YearsBoth21Phase 1United States
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231NCT00301834January 200516 December 2017Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)Not recruitingN/A21 YearsAll35Phase 2United States
232NCT00566488January 200520 May 2019Parathyroid and Thymus Transplantation in DiGeorge #931Parathyroid and Thymus Transplantation in DiGeorge Syndrome, #931DiGeorge Syndrome;Hypoparathyroidism;Complete DiGeorge SyndromeBiological: Thymus/Parathyroid TransplantationM. Louise MarkertFood and Drug Administration (FDA);National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbHNot recruitingN/A24 MonthsAll25Phase 1United States
233NCT00123916November 200419 February 2015BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' DiseaseBenznidazole Evaluation for Interrupting Trypanosomiasis - The BENEFIT TrialChagas Disease;Trypanosomiasis;Heart DiseaseDrug: Benznidazole;Drug: PlaceboPopulation Health Research InstituteCanadian Institutes of Health Research (CIHR);World Health Organization;Dante Pazzanese Institute;Hospital das Clinicas de Ribeirao Preto/USPRecruiting18 Years75 YearsBoth1000Phase 3Argentina;Bolivia;Brazil;Colombia;El Salvador;Canada
234NCT00146627September 200410 October 2016Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African TrypanosomiasisClinical Study Comparing the Nifurtimox-Eflornithine Combination With the Standard Eflornithine Regimen for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis in the Meningoencephalitic PhaseTrypanosomiasis, AfricanDrug: Eflornithine;Drug: NifurtimoxDrugs for Neglected DiseasesMedecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health Organization;Medecins Sans Frontieres, Netherlands;PNLTHA-DRC;;PNLTHA-RoC;Epicentre;Swiss Tropical & Public Health Institute;World Health OrganizationNot recruiting15 Years70 YearsBoth280Phase 3Congo, The Democratic Republic of the;Congo;The Democratic Republic of the Congo;Uganda
235NCT00168012September 200419 February 2015Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)An Open Study to Evaluate the Safety and Efficacy of IVIG-F10 in Patients With Primary Immunodeficiency Diseases (PID)Agammaglobulinemia;IgG Deficiency;Common Variable ImmunodeficiencyDrug: Immunoglobulins Intravenous (Human)CSL BehringNot recruiting3 Years70 YearsBoth42Phase 3
No.TrialIDDate_
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236NCT00168025September 200419 February 2015Efficacy and Safety of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)A Multicenter Study on the Efficacy, Safety and Pharmacokinetics of IgPro10 in Patients With Primary Immunodeficiency (PID)Agammaglobulinemia;IgG Deficiency;Common Variable ImmunodeficiencyDrug: Immunoglobulins Intravenous (Human)CSL BehringNot recruiting3 Years70 YearsBoth89Phase 3
237NCT00228852April 200419 February 2015IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell ImmunodeficiencyPhase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell ImmunodeficiencyT-Cell Immune Deficiency Diseases;Severe Combined ImmunodeficiencyDrug: Busulfan, Fludarabine and ATGEmory UniversityNot recruitingN/AN/ABothPhase 1/Phase 2United States
238NCT00578643March 20042 July 2018Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous DiseaseHLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Busulfan;Biological: Alemtuzumab;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Cyclosporine;Procedure: Stem Cell InfusionBaylor College of MedicineTexas Children's HospitalNot recruitingN/AN/AAll15Phase 2United States
239NCT00152100February 200419 February 2015Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency SyndromeTransplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency SyndromeSevere Combined ImmunodeficiencyProcedure: Stem cell transplant;Drug: Filgrastim, Alemtuzumab;Device: Miltenyi CliniMACSSt. Jude Children's Research HospitalNot recruitingN/A2 YearsBoth4Phase 1United States
240NCT00564759January 200419 February 2015Gene Therapy for Chronic Granulomatous DiseasePhase I/II Gene Therapy Study for X-Linked Chronic Granulomatous DiseaseGranulomatous Disease, ChronicDrug: retroviral SF71-gp91phox transduced CD34+ cellsJohann Wolfgang Goethe University HospitalsGerman Federal Ministry of Education and ResearchNot recruiting18 YearsN/AMale2Phase 1/Phase 2Germany
No.TrialIDDate_
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241NCT01279720October 200321 September 2015Gene Therapy ADA DeficiencyPhase I Gene Therapy Protocol for Adenosine Deaminase DeficiencyAdenosine Deaminase DeficiencyBiological: Intravenous infusion of transduced cellsGreat Ormond Street Hospital for Children NHS Foundation TrustNot recruitingN/A18 YearsBoth8Phase 1/Phase 2United Kingdom
242NCT00065390July 200319 February 2015Pyrimethamine to Treat Autoimmune Lymphoproliferative SyndromePilot (Phase I-II) Study of Pyrimethamine (Daraprim) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS)Autoimmune Disease;Lymphatic Disease;Lymphoproliferative DisorderDrug: PyrimethamineNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth8Phase 1United States
243NCT00803933February 200319 February 2015Trial of DB289 for the Treatment of Stage I African TrypanosomiasisPhase II b Trial of DB289 for the Treatment of Stage I African TrypanosomiasisAfrican TrypanosomiasisDrug: DB289;Drug: PentamidineImmtech Pharmaceuticals, IncBill and Melinda Gates FoundationNot recruiting15 Years50 YearsBoth111Phase 2Congo
244NCT00489658October 200219 February 2015Eflornithine + Nifurtimox Late-Stage Human African Trypanosomiasis (HAT)in West Nile, UgandaEfficacy and Safety of an Eflornithine + Nifurtimox Combination for Treatment of Late-Stage Human African Trypanosomiasis (HAT) in West Nile, UgandaTrypanosomiasis, AfricanDrug: Eflornithine plus Nifurtimox combination therapyEpicentreMedecins Sans FrontieresNot recruitingN/AN/ABoth31Phase 2/Phase 3Uganda
245NCT00220766August 200219 February 2015Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency PatientsIGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient PatientsImmunologic Deficiency Syndrome;Agammaglobulinemia;Severe Combined Immunodeficiency;Wiskott-Aldrich Syndrome;Common Variable ImmunodeficiencyDrug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified;Drug: Dextrose, 5% in WaterGrifols Therapeutics Inc.Not recruiting18 Years75 YearsBoth100Phase 3United States;Canada
No.TrialIDDate_
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246NCT00579709July 200229 July 2019Thymus Transplantation With ImmunosuppressionThymus Transplantation With Immunosuppression, #884DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete DiGeorge SyndromeBiological: Thymus Tissue for TransplantationM. Louise MarkertNational Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbHNot recruitingN/AN/AAll15Phase 1United States
247NCT00157079June 25, 200229 January 2018Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency DisordersA Clinical Investigation to Assess the Safety and Efficacy of Immune Globulin Intravenous (Human), 10% in Subjects With Primary Immunodeficiency DisordersPrimary Immunodeficiency Diseases (PID);Immune Thrombocytopenic Purpura (ITP);Kawasaki SyndromeBiological: Immune Globulin Intravenous (Human), 10%Baxalta now part of ShireNot recruiting24 MonthsN/AAllPhase 3United States
248NCT00161993June 13, 200226 August 2019Safety, Pharmacokinetic and Efficacy Study of a 10% Triple Virally Reduced Intravenous Immune Globulin Solution in Patients With Primary Immunodeficiency (Hypo- or Agammaglobulinemia)Prospective Open-Label Study of Pharmacokinetics, Efficacy and Safety of Immune Globulin Intravenous (Human), 10% TVR Solution in Patients With Hypo- or AgammaglobulinemiaPrimary Immunodeficiency Diseases (PID);Agammaglobulinemia;HypogammaglobulinemiaDrug: Immune Globulin Intravenous (Human), 10% TVR (Triple Virally Reduced) Solution;Drug: Gammagard S/D (Solvent/Detergent)Baxalta now part of ShireNot recruiting18 YearsN/AAllPhase 2Finland;Sweden
249NCT00176852June 20021 October 2018Stem Cell Transplant for HemoglobinopathyAllogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed ChimerismSickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond SyndromeDrug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusionMasonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramNot recruitingN/A50 YearsAll22Phase 2/Phase 3United States
250NCT01019876June 200219 February 2015Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
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251NCT00661401January 200219 February 2015Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous ImmunoglobulinSerum IgG Antibody to Streptococcus Pneumoniae, Haemophilus Influenzae Type b and Tetanus Toxoid in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin InfusionsCommon Variable Immunodeficiency;AgammaglobulinemiaBiological: gammaglobulinFederal University of São PauloCSL BehringNot recruiting2 Years75 YearsBoth5N/ABrazil
252NCT00028236December 10, 200116 December 2017Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)Ex Vivo Retroviral Gene Transfer For Treatment of X-Linked Severe Combined Immunodeficiency (XSCID)Severe Combined ImmunodeficiencyDrug: Gene-Transduced Autologous CD34+ Stem CellsNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting18 Months20 YearsAll3Phase 1United States
253NCT00576407November 200130 September 2019Thymus Transplantation in DiGeorge Syndrome #668Phase II Study of Thymus Transplantation in Complete DiGeorge Syndrome #668DiGeorge Syndrome;Complete Typical DiGeorge AnomalyBiological: Thymus Tissue for TransplantationM. Louise MarkertNational Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Enzyvant Therapeutics GmbHNot recruitingN/AN/AAll26Phase 2United States
254NCT00024934October 20013 October 2016B-Lymphocyte Stimulator (BLyS) To Treat Selective IgA DeficiencyA Phase I Dose Escalation Study of B-Lymphocyte Stimulator (BLyS) Administered Subcutaneously in Patients With Selective IgA DeficiencyIgA DeficiencyDrug: B-Lymphocyte Stimulator (BLyS)National Cancer Institute (NCI)Not recruitingN/AN/ABoth20Phase 1United States
255NCT00023192August 200119 February 2015Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of CareTreatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of CareChronic Granulomatous DiseaseDrug: T-Cell Depleted & CD34+Select/w/StemCell Enriched ProductNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth60Phase 3United States
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256NCT00802594August 200119 February 2015A Trial of DB289 for the Treatment of Stage I African TrypanosomiasisPhase II A Trial of DB289 for the Treatment of Stage I African TrypanosomiasisTrypanosomiasis, AfricanDrug: DB289Immtech Pharmaceuticals, IncBill and Melinda Gates FoundationNot recruiting16 YearsN/ABoth30Phase 2Angola;Congo
257NCT00018018June 20, 200111 November 2019Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) DeficiencyTreatment of SCID Due to ADA Deficiency With Autologous Cord Blood or Bone Marrow CD34+ Cells Transduced With a Human ADA GeneSevere Combined Immunodeficiency SyndromeDrug: CD34+ cells transduced with ADA retrovirNational Human Genome Research Institute (NHGRI)Not recruiting1 MonthN/AAll8Phase 1United States
258NCT00013689March 200119 February 2015Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative SyndromePilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS)Autoimmune Disease;Lymphoproliferative DisorderDrug: Fansidar (pyrimethamine and sulfadoxine)National Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth8Phase 1United States
259NCT00330148March 200119 February 2015Randomized Clinical Trial of Three Drug Combinations for Late-Stage Gambiense Human African TrypanosomiasisClinical Trial Comparing the Therapeutic Combinations Melarsoprol-Nifurtimox, Melarsoprol-Eflornithine and Eflornithine-Nifurtimox in the Treatment of Gambiense Human African Trypanosomiasis in the Meningo-Encephalitic PhaseTrypanosomiasis, AfricanDrug: melarsoprol 1.8 mg/kg/d, 10d + nifurtimox 15/20 mg/kg/d, 10d;Drug: melarsoprol 1.8 mg/kg/d, 10d + eflornithine 400 mg/kg/d, 7d;Drug: nifurtimox 15/20 mg/kg/d 10d + eflornithine 400 mg/kg/d 7dEpicentreMedecins Sans Frontieres;Embassy of France in Uganda;National Sleeping Sickness Control Program, UgandaNot recruitingN/AN/ABoth435Phase 3Uganda
260NCT00031486September 200019 October 2017Long Term Treatment of Herpes Simplex Encephalitis (HSE) With ValacyclovirA Phase III Double-Blind, Placebo-Controlled Trial of Long Term Therapy of Herpes Simplex Encephalitis (HSE): An Evaluation of Valacyclovir (CASG-204)EncephalitisDrug: Valacyclovir;Drug: PlaceboNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting12 YearsN/AAll91Phase 3United States;Canada;Sweden;United Kingdom
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261NCT00305708August 200019 February 2015Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)Not recruitingN/A17 YearsBoth40Phase 1/Phase 2United States
262NCT00176878June 200019 October 2017Stem Cell Transplant for Bone Marrow Failure SyndromesBone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure DisordersDiamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond SyndromeProcedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNot recruitingN/A35 YearsAll10Phase 2/Phase 3United States
263NCT00006054March 200019 February 2015Allogeneic Bone Marrow Transplantation in Patients With Primary ImmunodeficienciesImmunologic Deficiency Syndromes;Chediak-Higashi Syndrome;Common Variable Immunodeficiency;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Hyper IgM Syndrome;Severe Combined Immunodeficiency;Leukocyte Adhesion Deficiency Syndrome;Virus-Associated Hemophagocytic SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow TransplantationFairview University Medical CenterNot recruitingN/A35 YearsBothN/AUnited States
264NCT00006056March 200019 February 2015Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic DisordersChediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantationFairview University Medical CenterNot recruitingN/A55 YearsBoth40N/AUnited States
265NCT00001145October 199919 February 2015Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM SyndromeStudy of Immune Responses and Safety of Recombinant CD40 Ligand in Patients With X-Linked Hyper IgM SyndromeImmunoproliferative DisorderDrug: Bacteriophage;Drug: rhuCD40L;Drug: KLHNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth5Phase 2United States
No.TrialIDDate_
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266NCT00001905April 199919 February 2015Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type IInterferon Gamma Administration in Leukocyte Adhesion Deficiency Type ILeukocyte Adhesion Deficiency SyndromeDrug: Interferon gammaNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth5Phase 2United States
267NCT00004695September 19977 April 2015Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable ImmunodeficiencyCommon Variable ImmunodeficiencyDrug: PEG-interleukin-2Mount Sinai School of MedicineNot recruiting2 YearsN/ABoth48N/A
268NCT00008450August 11, 199726 August 2019Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantInduction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate MofetilAdenosine Deaminase Deficiency;Autosomal Recessive Disorder;Immune System Disorder;Purine-Nucleoside Phosphorylase Deficiency;Severe Combined Immunodeficiency;Severe Combined Immunodeficiency With Absence of T and B Cells;X-Linked Severe Combined ImmunodeficiencyProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Not recruitingN/AN/AAll6Phase 1United States
269NCT00001476June 1, 199516 December 2017Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-upGene Therapy Approach for Chronic Granulomatous DiseaseChronic Granulomatous Disease;Communicable DiseaseDrug: Gene Therapy Method for CGD;Device: Isolex 300i Magnetic Cell SelectorNational Institute of Allergy and Infectious Diseases (NIAID)Not recruiting5 YearsN/AAll14Phase 1United States
270NCT00004787December 199419 February 2015Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure SyndromesShwachman Syndrome;Fanconi's Anemia;Dyskeratosis Congenita;ThrombocytopeniaDrug: filgrastimNational Center for Research Resources (NCRR)James Whitcomb Riley Hospital for ChildrenNot recruitingN/AN/ABoth20Phase 2
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271NCT00001317May 199219 February 2015A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of ChildhoodA Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of ChildhoodChronic Granulomatous DiseaseDrug: interferon-gammaNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth100Phase 4United States
272NCT00001280January 199119 February 2015Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous DiseaseItraconazole for the Prevention of Fungal Infections in Chronic Granulomatous DiseaseMycosesDrug: itraconazoleNational Institute of Allergy and Infectious Diseases (NIAID)Not recruitingN/AN/ABoth100Phase 2United States
273NCT00001255September 199019 February 2015Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History StudyTreatment of Severe Combined Immunodeficiency Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency With Autologous Lymphocytes of CD34+ Cells Transduced With a Human ADA Gene: A Natural History StudySevere Combined ImmunodeficiencyDrug: ADA PBSC;Drug: ADA Umbilical Cord Blood Cells;Drug: Transduced LymphocytesNational Human Genome Research Institute (NHGRI)Not recruitingN/AN/ABoth10N/AUnited States

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