Cd34+    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
65原発性免疫不全症候群30

65. 原発性免疫不全症候群 [臨床試験数:413,薬物数:581(DrugBank:97),標的遺伝子数:68,標的パスウェイ数:202
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.
30 / 413 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-002343-14-NL
(EUCTR)
25/09/202026/07/2019GENE THERAPY IN CHILDREN WITH RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCYPHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1);Therapeutic area: Diseases [C] - Immune System Diseases [C20]Product Name: RAG1 LV CD34+ cells
Product Code: RAG1 LV CD34+ cells
Leiden University Medical CenterNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 1;Phase 2Netherlands
2EUCTR2020-000517-33-GB
(EUCTR)
14/07/202021/02/2020Gene 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 GeneGene 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 Gene Leukocyte 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
INN or Proposed INN: CD34+CELLS
Other descriptive name: CD34+CELLS
Rocket Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
9Phase 1;Phase 2United States;Spain;United Kingdom
3EUCTR2018-003842-18-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
4NCT03601286
(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
5EUCTR2018-002680-26-ES
(EUCTR)
27/11/201802/08/2018Gene 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 Gene Leukocyte 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
INN or Proposed INN: CD34+CELLS
Other descriptive name: CD34+CELLS
Rocket Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
2Phase 1Spain
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2018-000673-68-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
7NCT03311503
(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
8NCT03765632
(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
9EUCTR2017-001275-23-GB
(EUCTR)
21/09/201703/07/2017A clinical trial to study the effects of genetically modified patients' CD34+ cellsEfficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two.
MedDRA version: 20.1;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells
Product Code: OTL-101
INN or Proposed INN: There is no recommended INN
Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene
Great Ormond Street Hospital for Children NHS TrustNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 2United Kingdom
10NCT02999984
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT01512888
(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
12NCT02234934
(ClinicalTrials.gov)
October 29, 20154/9/2014Study 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)Active, not recruiting23 MonthsN/AMale10Phase 1;Phase 2United States
13EUCTR2014-000274-20-GB
(EUCTR)
14/05/201412/03/2014Long-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 2.0 Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiencycaused by mutations in a single gene ,the Wiskott-Aldrich SyndromeProtein (WASP). WAS is characterised by micro-thrombocytopenia,recurrent infections,eczema and associated with a high incidence ofauto-immunity and of lymphoid malignancies. Over 150 uniquemutations in the WAS gene have been identified.Loss-of-functionmutations in this gene have widespread consequences on hematopoieticlineages.
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
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
10Phase 2United Kingdom
14EUCTR2012-000242-35-DE
(EUCTR)
23/12/201329/04/2013Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease (X-CGD)A 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 patients X-linked Chronic Granulomatous Disease
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] - Immune System Diseases [C20]
Product Name: Suspension of autologous CD34+cells transduced with the G1XCGD viral vector
Product Code: G1XCGD transduced CD34+ cells
Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR
GENETHONNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
20Phase 1;Phase 2France;Germany;United Kingdom;Switzerland
15NCT01852071
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT01966367
(ClinicalTrials.gov)
March 201317/10/2013CD34+ (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 GeorgeNULLActive, not recruitingN/A40 YearsAll37Phase 1;Phase 2United States
17EUCTR2012-000242-35-GB
(EUCTR)
10/01/201319/07/2012Gene 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 withX-Linked Chronic Granulomatous Disease - Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients X-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
Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
11Phase 1;Phase 2France;Germany;Switzerland;United Kingdom
18NCT01380990
(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
19NCT01306019
(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
20EUCTR2010-024253-36-GB
(EUCTR)
03/08/201209/05/2012A clinical trial to study the effects of genetically modified patients' CD34+ cellsPhase 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 autologus CD34+ cells from ADA-deficient individuals - LV Gene Therapy for ADA Deficiency Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two.
MedDRA version: 20.0;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000012248;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells
Product Code: transduced patient CD34+ cells
INN or Proposed INN: EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells
Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene
Great Ormond Street Hospital for Children NHS TrustNULLNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT03315078
(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
22NCT01856582
(ClinicalTrials.gov)
October 201015/5/2013CD34+ 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 CenterTerminatedN/A35 YearsAll23Phase 2United States
23EUCTR2007-004308-11-GB
(EUCTR)
11/01/201006/07/2009Gene therapy for WASPHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPYFOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0 Wiskott-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
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
Phase 1;Phase 2United Kingdom
24NCT00794508
(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
25NCT00564759
(ClinicalTrials.gov)
January 200426/11/2007Gene 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 ResearchActive, not recruiting18 YearsN/AMale2Phase 1;Phase 2Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT00028236
(ClinicalTrials.gov)
December 10, 200117/12/2001Stem 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)NULLCompleted18 Months20 YearsAll3Phase 1United States
27NCT00023192
(ClinicalTrials.gov)
August 200129/8/2001Treatment 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)NULLCompletedN/AN/ABoth60Phase 3United States
28NCT00018018
(ClinicalTrials.gov)
June 20, 200127/6/2001Gene 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)NULLCompleted1 MonthN/AAll8Phase 1United States
29NCT00001255
(ClinicalTrials.gov)
September 19903/11/1999Gene 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)NULLCompletedN/AN/ABoth10N/AUnited States
30EUCTR2009-011152-22-FR
(EUCTR)
09/12/2010Phase 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 WAS Phase 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
GENETHONNULLNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2France