Found 1367 matching trials
CIBMTR Research Database
N/AThe primary purpose of the Research Database is to have a comprehensive sou...
Location
Center for International Blood and Marrow Transplant Research
Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
PHASE1Background:
X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a ...
Status
Enrolling By Invitation
Location
National Institutes of Health Clinical Center
Clinical Characterization on PDE6A-related Retinitis Pigmentosa in Preparation to a Gene Therapy Trial
N/AMutations in the PDE6A gene - encoding the -subunit of the rod cGMP-phospho...
Location
Institute for Ophthalmic Research, University Tübingen, Germany
Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects
PHASE2This trial is designed to expand the currently available data on the safety...
Location
ECOGENE-21 Clinical Trial Center / Centre de santé et de services sociaux de Chicoutimi
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
PHASE1This clinical study is in participants with Amyotrophic Lateral Sclerosis a...
Location
Macquarie University Hospital
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
PHASE1The purpose of this study is to evaluate the safety and efficacy of exogeno...
Location
David Geffen School of Medicine at UCLA
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
PHASE1The purpose of this clinical trial is to examine the safety of gene therapy...
Location
Hadassah Medical Organization
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
PHASE2The objective of this clinical study is to select the optimal dose and eval...
Location
Beijing Tongren Hospital, Capital Medical University
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
PHASE1This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expan...
Status
Active Not Recruiting
Location
AllerVie Clinical Research
Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)
PHASE1Chronic Granulomatous Disease (CGD) is a rare inherited disorder in which p...
Location
Great Ormond Street Hospital for Children NHS Trust