65. Primary immunodeficiency
413 clinical trials,   581 drugs   (DrugBank: 97 drugs),   68 drug target genes,   202 drug target pathways

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
42 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04370795
(ClinicalTrials.gov)
December 17, 202030/4/2020Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusMatched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and SirolimusSevere Combined Immunodeficiency (SCID)Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI)National Institute of Allergy and Infectious Diseases (NIAID)NULLEnrolling by invitation3 Years40 YearsAll30Phase 1;Phase 2United States
2NCT03597594
(ClinicalTrials.gov)
December 202019/6/2018Haplocompatible 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 HospitalNULLRecruiting2 MonthsN/AAll42Phase 1;Phase 2United States
3NCT04339777
(ClinicalTrials.gov)
September 22, 20208/4/2020Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency DiseasesA Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immunodeficiency DiseasesLymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency DisordersDrug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irradiation;Procedure: Allogeneic HSCT;Drug: Tacrolimus (Tacro);Drug: Mycophenolate mofetil (MMF);Drug: Cyclophosphamide (Cytoxan)National Cancer Institute (NCI)NULLRecruiting4 Years69 YearsAll56Phase 2United States
4NCT03910452
(ClinicalTrials.gov)
October 28, 20199/4/2019Haploidentical 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)NULLRecruiting4 Years65 YearsAll30Early Phase 1United States
5EUCTR2018-003842-18-IT
(EUCTR)
08/01/201919/11/2018Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS)A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. Wiskott-Aldrich Syndrome
MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: OTL-103 Dispersion for Infusion
Product Code: OTL-103
INN or Proposed INN: Other hematological Agents
Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
Other descriptive name: NA
Trade Name: Fludarabina Accord
INN or Proposed INN: FLUDARABINE
Other descriptive name: NA
Trade Name: MabThera
INN or Proposed INN: RITUXIMAB
Other descriptive name: NA
Trade Name: Mozobil,
INN or Proposed INN: plerixafor
Other descriptive name: PLERIXAFOR
Trade Name: MYELOSTIM
Product Name: granulocyte colony stimulating factor (G-CSF)
INN or Proposed INN:
Orchard Therapeutics Ltd.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
6Phase 3Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03601286
(ClinicalTrials.gov)
December 21, 201822/2/2018Lentiviral 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 TrustNULLRecruiting8 Weeks5 YearsMale5Phase 1United Kingdom
7NCT03619551
(ClinicalTrials.gov)
October 22, 201816/7/2018Conditioning 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, MDNULLRecruitingN/A2 YearsAll64Phase 2United States
8EUCTR2018-000673-68-GB
(EUCTR)
09/10/201827/07/2018 A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined ImmunodeficiencyPhase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening.
MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Great Ormond Street Hospital for Children NHS TrustNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
5Phase 1United Kingdom
9NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning 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 ResearchRecruiting3 Months39 YearsAll40Phase 1;Phase 2United States
10NCT03538899
(ClinicalTrials.gov)
May 31, 20183/5/2018Autologous 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 FranciscoNULLRecruiting2 MonthsN/AAll15Phase 1;Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03311503
(ClinicalTrials.gov)
January 19, 201812/10/2017Phase 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 WilliamsNULLRecruitingN/A5 YearsMale10Phase 1;Phase 2United States;United Kingdom
12JPRN-UMIN000030806
2018/01/1715/01/2018A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome Wiskott-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 DevelopmentNULLRecruitingNot applicableNot applicableMale3Phase 1;Phase 2Japan
13NCT03765632
(ClinicalTrials.gov)
January 3, 20188/8/2018Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDEfficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase DeficiencySevere Combined Immunodeficiency Due to ADA DeficiencyGenetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsRecruitingN/A17 YearsAll10Phase 1;Phase 2United Kingdom
14JPRN-jRCTs031180398
30/12/201720/03/2019A phase II study of RIC-SCT for CGDReduced 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 disease
Primary immunodeficiency;D006105
Conditioning regimen with targeted-busulfan and fludarabin
Total body irradiation (3Gy) at day -1
Stem cell transplantation at day 0
Kato MotohiroNULLRecruitingNot applicable< 25age oldBoth22Phase 2None (Japan only);Japan
15JPRN-UMIN000030647
2017/12/3031/12/2017Reduced 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 DevelopmentNULLRecruitingNot applicable25years-oldMale and Female22Phase 2Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT02999984
(ClinicalTrials.gov)
December 16, 201619/12/2016Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDEfficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA DeficiencySevere Combined Immunodeficiency Due to ADA DeficiencyGenetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG-ADA ERTOrchard TherapeuticsCalifornia Institute for Regenerative Medicine (CIRM);University of California, Los AngelesCompletedN/A17 YearsAll10Phase 1;Phase 2United States
17NCT01512888
(ClinicalTrials.gov)
August 17, 201613/1/2012Gene 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
18JPRN-UMIN000022688
2016/06/1013/06/2016A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease 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 DevelopmentNULLRecruitingNot applicable25years-oldMale and Female9Not selectedJapan
19NCT02629120
(ClinicalTrials.gov)
December 17, 201510/12/2015High 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: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting4 Years65 YearsAll50Phase 1;Phase 2United States
20EUCTR2013-005508-33-IT
(EUCTR)
13/07/201521/05/2015Comparison 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 diseas Male 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: 18.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850
MedDRA version: 18.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870
MedDRA version: 18.0;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)
INN or Proposed INN: TREOSULFAN
Trade Name: Ovastat 5000 (Treosulfan injection)
INN or Proposed INN: TREOSULFAN
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
medac GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT02349906
(ClinicalTrials.gov)
April 201526/1/2015Treosulfan-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 HealthActive, not recruitingN/A17 YearsAll100Phase 2Czechia;Germany;Italy;Poland;Austria;Czech Republic
22EUCTR2013-005508-33-PL
(EUCTR)
17/11/201408/08/2014Comparison 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 diseas Male 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;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870
MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
medac GmbHNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
23EUCTR2013-005508-33-CZ
(EUCTR)
12/11/201429/07/2014Comparison 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 diseas Male 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;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870
MedDRA version: 20.0;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 mg, powder for solution for infusion
Product Name: Ovastat 1000
INN or Proposed INN: TREOSULFAN
Trade Name: Ovastat 5000 mg, powder for solution for infusion
Product Name: Ovastat 5000
INN or Proposed INN: TREOSULFAN
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
medac Gesellschaft für klinische Spezialpräparate mbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
24NCT02282904
(ClinicalTrials.gov)
October 23, 20144/11/2014Haploidentical 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)NULLTerminated2 Years65 YearsAll7Phase 1;Phase 2United States
25EUCTR2013-005508-33-DE
(EUCTR)
29/09/201406/06/2014Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases Clinical 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 diseas Male 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;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870
MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
medac Gesellschaft für klinische Spezialpräparate mbHNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2013-005508-33-AT
(EUCTR)
08/08/201408/07/2014Comparison 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 diseas Male 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: 19.1;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850
MedDRA version: 19.1;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870
MedDRA version: 19.1;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)
INN or Proposed INN: TREOSULFAN
Trade Name: Ovastat 5000 (Treosulfan injection)
INN or Proposed INN: TREOSULFAN
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
medac Gesellschaft fuer klinische Spezialpräparate mbHNULLNot RecruitingFemale: yes
Male: yes
100Phase 2Czech Republic;Poland;Austria;Germany;Italy
27NCT01852071
(ClinicalTrials.gov)
August 2, 20137/5/2013Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA GeneAutologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)ADA-SCIDGenetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG-ADA ERTOrchard TherapeuticsNational Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los AngelesCompleted1 Month17 YearsAll20Phase 1;Phase 2United States
28NCT01380990
(ClinicalTrials.gov)
November 15, 201223/6/2011Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyPhase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient IndividualsAdenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID)Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsCompletedN/A15 YearsAll36Phase 1;Phase 2United Kingdom
29NCT01306019
(ClinicalTrials.gov)
September 25, 201226/2/2011Lentiviral 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 Immune Deficiency (XSCID)Drug: Palifermin;Drug: Busulfan;Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1a-hyc-OPT vectorNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting2 Years40 YearsMale30Phase 1;Phase 2United States
30NCT01652092
(ClinicalTrials.gov)
September 4, 201225/7/2012Allogeneic 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: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNAMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A50 YearsAll30N/AUnited States
No.TrialIDDate_
enrollment
Date_
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Public_titleScientific_titleConditionInterventionPrimary_
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31NCT03315078
(ClinicalTrials.gov)
April 201216/10/2017Lentiviral 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)NULLRecruiting2 Years40 YearsAll13Phase 1;Phase 2United States
32NCT01338675
(ClinicalTrials.gov)
January 20114/3/2011Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)Chronic Granulomatous DiseaseDrug: BusulfanSeoul National University HospitalNULLRecruitingN/AN/ABoth5Phase 1;Phase 2Korea, Republic of
33NCT00794508
(ClinicalTrials.gov)
November 200819/11/2008MND-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)Completed1 Month18 YearsAll10Phase 2United States
34NCT00885833
(ClinicalTrials.gov)
February 200721/4/2009Study 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 OncologyNULLCompleted1 Year25 YearsBoth5Phase 1;Phase 2Korea, Republic of
35NCT00301834
(ClinicalTrials.gov)
January 20059/3/2006Alemtuzumab, 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)CompletedN/A21 YearsAll35Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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PhaseCountries
36NCT00228852
(ClinicalTrials.gov)
April 200427/9/2005IMM 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 UniversityNULLCompletedN/AN/ABothPhase 1;Phase 2United States
37NCT00578643
(ClinicalTrials.gov)
March 200419/12/2007Matched 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 MedicineNULLCompletedN/AN/AAll15Phase 2United States
38NCT00176852
(ClinicalTrials.gov)
June 200212/9/2005Stem 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 ProgramCompletedN/A50 YearsAll22Phase 2;Phase 3United States
39NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, 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)CompletedN/A17 YearsBoth40Phase 1;Phase 2United States
40NCT00176878
(ClinicalTrials.gov)
June 200012/9/2005Stem 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 MinnesotaNULLCompletedN/A35 YearsAll10Phase 2;Phase 3United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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size
PhaseCountries
41NCT00006056
(ClinicalTrials.gov)
March 20005/7/2000Pilot 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 CenterNULLActive, not recruitingN/A55 YearsBoth40N/AUnited States
42NCT00006054
(ClinicalTrials.gov)
March 20005/7/2000Allogeneic 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 CenterNULLTerminatedN/A35 YearsBothN/AUnited States