RP-L201 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
65Primary immunodeficiency4

65. Primary immunodeficiency


Clinical trials : 482 Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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PhaseCountries
1EUCTR2020-000517-33-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
2NCT03812263
(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
3NCT03825783
(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
4EUCTR2018-002680-26-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