65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details


臨床試験数 : 500 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217

  
45 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT05071222
(ClinicalTrials.gov)
January 202327/8/2021Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)A Phase 1/2 Open Label Non Randomized Study, Multicentric, Single Arm Evaluating the Safety and Efficacy of Gene Therapy of the Severe Combined Immunodeficiency (SCID) Caused by Mutations in the Human DCLRE1C Gene (Artemis) by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced ex Vivo With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNAArtemis (DCLRE1C ) Deficient Severe Combined ImmunodeficiencyGenetic: ARTEGENE drug productAssistance Publique - Hôpitaux de ParisNULLNot yet recruitingN/A1 YearAll5Phase 1/Phase 2France
2NCT04797260
(ClinicalTrials.gov)
July 23, 202111/3/2021Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCIDPhase I/II Clinical Trial of Autologous Hematopoietic Stem Cell Gene Therapy in RAG1-Deficient Severe Combined ImmunodeficiencySevere Combined Immunodeficiency Due to RAG1 DeficiencyGenetic: Gene therapyLeiden University Medical CenterZonMw: The Netherlands Organisation for Health Research and Development;Horizon 2020 - European CommissionRecruiting8 Weeks24 MonthsAll10Phase 1/2Netherlands
3EUCTR2019-002343-14-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
4EUCTR2020-000517-33-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
5NCT04049084
(ClinicalTrials.gov)
September 26, 20196/8/2019An 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)University of California, Los AngelesGreat Ormond Street Hospital for Children NHS Foundation TrustEnrolling by invitationN/AN/AAll70United States;United Kingdom
6NCT03812263
(ClinicalTrials.gov)
August 30, 201918/1/2019A 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.California Institute for Regenerative Medicine (CIRM)Active, not recruiting3 MonthsN/AAll9Phase 1/Phase 2United States;Spain;United Kingdom
7NCT03825783
(ClinicalTrials.gov)
April 15, 201925/1/2019A 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.NULLRecruiting3 MonthsN/AAll2Phase 1Spain
8NCT03837483
(ClinicalTrials.gov)
January 21, 20198/2/2019A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich SyndromeA Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)Wiskott-Aldrich SyndromeGenetic: OTL-103Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingN/A65 YearsAll10Phase 3United States;Italy
9EUCTR2018-003842-18-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
10NCT03601286
(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
11EUCTR2018-002680-26-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.NULLNot RecruitingFemale: yes
Male: yes
2Phase 1Spain
12EUCTR2018-000673-68-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]
Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells
INN or Proposed INN: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells
Great Ormond Street Hospital for Children NHS TrustNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
5 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
13NCT03645486
(ClinicalTrials.gov)
July 1, 201824/7/2018Lentiviral Gene Therapy for CGDLentiviral Gene Therapy for Chronic Granulomatous Disease (CGD)Chronic Granulomatous DiseaseGenetic: Infusion of lentiviral TYF-CGD-modified autologous stem cellsShenzhen Geno-Immune Medical InstituteNULLRecruitingN/AN/AAll10N/AChina
14NCT03538899
(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/AAll25Phase 1/Phase 2United States
15JPRN-UMIN000030806
2018/01/1715/01/2018A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich SyndromeA phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - 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 DevelopmentNULLComplete: follow-up continuingNot applicableNot applicableMale3Phase 1,2Japan
16EUCTR2017-001731-39-IT
(EUCTR)
12/10/201702/07/2020Retroviral insertion site methodology studyMethodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy - Retroviral insertion site methodology study Adenosine deaminase (ADA) deficiency severe combined immunodeficiency
MedDRA version: 20.1;Level: LLT;Classification code 10066367;Term: Adenosine deaminase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Strimvelis
Product Name: Strimvelis
Product Code: [NA]
Orchard Therapeutics (Europe) LtdNULLNot RecruitingFemale: yes
Male: yes
15Phase 4Turkey;Switzerland;Italy
17NCT03478670
(ClinicalTrials.gov)
March 31, 201723/3/2018Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated With Strimvelis (Previously GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and EffectivenessImmunologic Deficiency SyndromesGenetic: StrimvelisOrchard TherapeuticsNULLEnrolling by invitationN/AN/AAll50Italy
18NCT02757911
(ClinicalTrials.gov)
March 201620/4/2016Gene Therapy for X-linked Chronic Granulomatous DiseaseA Phase I/II, Non Randomized, Monocentric Open-label Study of Autologous CD34+ Cells Transduced With the G1XCGD Lentiviral Vector in Patients With X-Linked Chronic Granulomatous DiseaseX-Linked Chronic Granulomatous DiseaseGenetic: X vivo gene therapyGenethonNULLActive, not recruiting24 MonthsN/AMale3Phase 1/Phase 2France
19NCT02333760
(ClinicalTrials.gov)
September 201428/10/2014Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich SyndromeLong 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 (GTG002-07 and GTG003-08).Wiskott-Aldrich SyndromeGenetic: Autologous CD34+ cells transduced with WASP lentiviral vectorGenethonNULLActive, not recruitingN/AN/AMale10Phase 1/Phase 2France;United Kingdom
20EUCTR2014-000274-20-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
21EUCTR2012-000242-35-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 1/2France;Germany;United Kingdom;Switzerland
22NCT01906541
(ClinicalTrials.gov)
July 201315/7/2013Gene Therapy for X-CGDA Phase I/II Gene Therapy Trial for X-CGD With a SIN Gammaretroviral VectorX-linked Chronic Granulomatous DiseaseGenetic: ex-vivo gene-therapyHubert Serve, Prof., MDNULLRecruiting18 YearsN/ABoth5Phase 1/Phase 2Germany
23NCT01855685
(ClinicalTrials.gov)
June 24, 201314/5/2013Gene Therapy 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 DiseaseX-Linked Chronic Granulomatous DiseaseGenetic: X vivo gene therapyGenethonNULLActive, not recruiting6 MonthsN/AMale3Phase 1/Phase 2United Kingdom;Germany;Switzerland
24EUCTR2012-001725-26-DE
(EUCTR)
12/03/201318/12/2012A 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-CGD Chronic 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
INN or Proposed INN: G1XCG
Other descriptive name: Genetically modified autologous blood stem cells
Johann Wolfgang Goethe-UniversityNULLNot RecruitingFemale: yes
Male: yes
5Phase 1;Phase 2Germany
25EUCTR2012-000242-35-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
26NCT01380990
(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
27EUCTR2010-024253-36-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
28NCT01381003
(ClinicalTrials.gov)
November 201123/6/2011Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)Phase I/II Gene Therapy Protocol for X-Linked Chronic Granulomatous DiseaseGranulomatous Disease, Chronic, X-linked, VariantGenetic: pCCLchimGp91s lentiviral vector transduced CD34+ cells infusionGreat Ormond Street Hospital for Children NHS Foundation TrustNULLWithdrawnN/A30 YearsMale0Phase 1/Phase 2United Kingdom
29NCT01347346
(ClinicalTrials.gov)
May 20113/5/2011Gene Therapy for WASPhase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeGenetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS geneGenethonHôpital Necker-Enfants MaladesCompletedN/AN/AMale5Phase 1/Phase 2France
30NCT01175239
(ClinicalTrials.gov)
April 201129/7/2010Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)Gene Therapy for SCID-X1 Using a Self-inactivating (SIN) Gammaretroviral VectorX-linked Severe Combined ImmunodeficiencyGenetic: Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.preGreat Ormond Street Hospital for Children NHS Foundation TrustNULLActive, not recruitingN/A16 YearsMale1N/AUnited Kingdom
31NCT01347242
(ClinicalTrials.gov)
March 20113/5/2011Gene Therapy for Wiskott-Aldrich Syndrome (WAS)Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeGenetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS geneGenethonGreat Ormond Street Hospital for Children NHS Foundation Trust;Institute of Child HealthCompletedN/AN/AMale6Phase 1/Phase 2United Kingdom
32NCT01515462
(ClinicalTrials.gov)
April 20, 201023/12/2011Gene Therapy for Wiskott-Aldrich SyndromeA Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich SyndromeWiskott-Aldrich Syndrome (WAS)Genetic: OTL-103Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingN/AN/AAll8Phase 1/Phase 2Italy
33EUCTR2009-017346-32-IT
(EUCTR)
15/03/201026/04/2010HAEMATOPOIETIC STEM CELL GENE THERAPYA PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS Wiskott-Aldrich Syndrom
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]
Trade Name: FLUDARABINA TEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACONCINO DI VETRO DA 2 ML
Product Name: na
Product Code: [na]
INN or Proposed INN: FLUDARABINA
Other descriptive name: FLUDARABINA
Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO
Product Name: PLERIXAFOR
Product Code: [na]
INN or Proposed INN: PLERIXAFOR
Other descriptive name: Plerixafor
Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML
Product Name: na
Product Code: [na]
INN or Proposed INN: LENOGRASTIM
Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML
Product Name: RITUXIMAB
Product Code: [na]
INN or Proposed INN: RITUXIMAB
Other descriptive name: RITUXIMAB
Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI
Product Name: na
Product Code: [na]
Orchard Therapeutics (Europe) LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
8Phase 1;Phase 2Italy
34EUCTR2007-000684-16-GB
(EUCTR)
21/01/201017/09/2009Gene 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-X1 X-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
Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector transduced cells
Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector transduce
Great Ormond Street Hospital NHS Trust / University College London - Institute of Child HealthNULLNot RecruitingFemale: no
Male: yes
10Phase 1;Phase 2United Kingdom
35EUCTR2007-004308-11-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
36NCT00579137
(ClinicalTrials.gov)
October 200719/12/2007Allogeneic 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 HospitalTerminatedN/AN/AAll3Phase 1/Phase 2United States
37NCT00778882
(ClinicalTrials.gov)
January 200721/10/2008Gene 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.NULLActive, not recruitingN/AN/AMale2Phase 1/Phase 2Korea, Republic of
38NCT00394316
(ClinicalTrials.gov)
October 30, 200631/10/2006Gene 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)NULLTerminated3 Years55 YearsMale3Early Phase 1United States
39NCT00927134
(ClinicalTrials.gov)
June 200422/6/2009Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in ChildrenPhase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in ChildrenChronic Granulomatous DiseaseGenetic: retroviral SF71-gp91phox transduced CD34+ cellsUniversity of ZurichGoethe UniversityCompleted1 Year18 YearsMale2Phase 1/Phase 2Switzerland
40NCT00564759
(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
41NCT01279720
(ClinicalTrials.gov)
October 200318/1/2011Gene 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 TrustNULLCompletedN/A18 YearsBoth8Phase 1/Phase 2United Kingdom
42NCT00028236
(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
43NCT00001476
(ClinicalTrials.gov)
June 1, 19953/11/1999Gene 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)NULLCompleted5 YearsN/AAll14Phase 1United States
44NCT00599781
(ClinicalTrials.gov)
March 19928/1/2008Gene Therapy for ADA-SCIDTreatment of ADA-SCID by Gene Therapy on Somatic CellsSevere Combined Immunodeficiency SyndromeGenetic: gene transduced PBL and/or gene transduced HSCIRCCS San RaffaeleFondazione TelethonCompletedN/AN/ABoth8Phase 1/Phase 2NULL
45EUCTR2009-011152-22-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