Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy (DMD)
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|ClinicalTrials.gov Identifier: NCT03375164|
Recruitment Status : Active, not recruiting
First Posted : December 15, 2017
Last Update Posted : November 10, 2020
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Genetic: SRP-9001||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7. Micro-dystrophin (microDys-IV-001)|
|Actual Study Start Date :||January 4, 2018|
|Estimated Primary Completion Date :||April 2023|
|Estimated Study Completion Date :||April 2023|
Experimental: Cohort A
Patients between 3 months to 3 years of age, will receive intravenous SRP-9001.
Single IV infusion of SRP-9001.
Experimental: Cohort B
Patients between 4 to 7 years of age, will receive intravenous SRP-9001.
Single IV infusion of SRP-9001.
- Safety Based on Number of Participants with Adverse Events. [ Time Frame: From baseline up to 5 years ]AEs will be monitored and scored for severity and relatedness to the study article.
- Cohort A : Gross Motor Subtest Scaled (Bayley-III) score [ Time Frame: Screening, Day 30-3 Years ]Gross Motor Scaled Score measures motor development. The Bayley-III Gross Motor Subtest will be scored for Cohort A on every follow up visit starting at Day 30 through 3 years. Any patient that is 43-47 months of age, inclusive, at time of screening will have the scaled score calculated compared to normative data for 42 month old children. The Bayley-III provides normative data for children 1-42 months of age.
- Cohort B: Physical Therapy Assessments The 100 Meter Timed Test (100m) [ Time Frame: Screening, Day 30-5 Years ]The 100m will be the primary motor outcome for Cohort B.
- Micro-dystrophin Gene Expression Quantification by Immunofluorescence [ Time Frame: Screening and Day 90 ]Micro-dystrophin gene expression levels will be quantified by immunofluorescence and compared in pre and post muscle biopsies.
- Micro-dystrophin Gene Expression Quantification by Western Blot [ Time Frame: Screening and Day 90 ]Micro-dystrophin gene expression levels will be quantified by western blot analysis and compared in pre and post muscle biopsies.
- Physical Therapy Assessments North Star Ambulatory Assessment (NSAA) [ Time Frame: Screening, Day 30-5 Years ]The North Star Ambulatory Assessment (NSAA) will be an exploratory outcome initiated for Cohort A as soon as the child is four years of age and for cohort B. The NSAA measures the quality of ambulation in young boys with Duchenne Muscular Dystrophy.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03375164
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|