|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
2 December 2024 |
|
Main ID: |
NCT03375164 |
|
Date of registration:
|
04/12/2017 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
|
|
Scientific title:
|
Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001) |
|
Date of first enrolment:
|
January 4, 2018 |
|
Target sample size:
|
4 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT03375164 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 1/Phase 2
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Medical Director |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Sarepta Therapeutics, Inc. |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Cohort A participants: 3 months to 3 years of age, inclusive
- Cohort B participants: 4 to 7 years of age, inclusive
- Definitive diagnosis of DMD based on documented clinical findings and prior genetic
testing.
- Ability to cooperate with motor assessment testing.
- Cohort A participants: No previous treatment with corticosteroids.
- Cohort B participants: Stable dose equivalent of oral corticosteroids for at least
12 weeks prior to screening and the dose is expected to remain constant (except for
potential modifications to accommodate changes in weight) throughout the first year
of the study.
- Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (inclusive).
Exclusion Criteria:
- Exposure to gene therapy, investigational medication, or any treatment designed to
increase dystrophin expression within protocol specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Presence of any other clinically significant illness, medical condition, or
requirement for chronic drug treatment that in the opinion of the Investigator
creates unnecessary risk for gene transfer.
Other inclusion or exclusion criteria could apply.
Age minimum:
3 Months
Age maximum:
7 Years
Gender:
Male
|
|
Health Condition(s) or Problem(s) studied
|
|
Duchenne Muscular Dystrophy
|
|
Intervention(s)
|
|
Genetic: delandistrogene moxeparvovec
|
|
Primary Outcome(s)
|
|
Number of Participants With Adverse Events (AEs)
[Time Frame: Up to 5 years]
|
|
Secondary Outcome(s)
|
|
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot
[Time Frame: Baseline, Day 90]
|
|
Change From Baseline at Year 5 in the 100 Meter Timed Test
[Time Frame: Baseline, Year 5]
|
|
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
[Time Frame: Baseline, Day 90]
|
|
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity
[Time Frame: Baseline, Day 90]
|
|
Secondary ID(s)
|
|
SRP-9001-101
|
|
2021-000077-83
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|