Molecular genetic services
Total Trials
16
As Lead Sponsor
13
As Collaborator
3
Total Enrollment
603
NCT01344798
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
Phase: Phase 1
Role: Lead Sponsor
Start: Nov 30, 2006
Completion: Jun 30, 2010
NCT01347242
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Phase: Phase 1/2
Start: Mar 31, 2011
Completion: Nov 13, 2019
NCT01347346
Gene Therapy for WAS
Start: May 31, 2011
Completion: Jan 9, 2017
NCT01380964
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
Phase: N/A
Start: Jun 30, 2011
Completion: Dec 31, 2015
NCT01385917
Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
Start: Oct 31, 2011
Completion: Dec 31, 2018
NCT01855685
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
Start: Jun 24, 2013
Completion: Sep 30, 2032
NCT02057705
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
Role: Collaborator
Start: Feb 28, 2014
Completion: Jun 26, 2017
NCT02333760
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Start: Sep 30, 2014
Completion: Oct 31, 2032
NCT02302690
Immunity Against AAV in Crigler Najjar Patient
Start: Nov 30, 2014
Completion: Jun 30, 2017
NCT02234934
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Start: Oct 29, 2015
Completion: Dec 1, 2024
NCT02757911
Gene Therapy for X-linked Chronic Granulomatous Disease
Start: Mar 31, 2016
Completion: Jun 30, 2034
NCT03466463
Gene Therapy for Severe Crigler Najjar Syndrome
Start: Mar 19, 2018
Completion: Mar 30, 2030
NCT03882827
Natural History of Duchenne Muscular Dystrophy
Start: Dec 19, 2019
Completion: Jun 30, 2023
NCT03842878
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Start: Feb 19, 2020
Completion: Dec 30, 2023
NCT04940572
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
Phase: Phase 2
Start: Nov 26, 2021
Completion: Dec 1, 2025
NCT06518005
Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
Start: Nov 8, 2024
Completion: Sep 30, 2030
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