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Trial record 9 of 104 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03769116
Recruitment Status : Not yet recruiting
First Posted : December 7, 2018
Last Update Posted : December 7, 2018
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: SRP-9001 Drug: Placebo Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week Extension
Estimated Study Start Date : December 18, 2018
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2021


Arm Intervention/treatment
Experimental: SRP-9001
Single IV infusion of SRP-9001.
Drug: SRP-9001
Single IV infusion of SRP-9001

Placebo Comparator: Placebo
Placebo IV infusion 10 mL/kg.
Drug: Placebo
Single IV infusion of placebo




Primary Outcome Measures :
  1. Incidence of Serious Adverse Events (SAEs) [ Time Frame: Up to 144 weeks ]
  2. Incidence of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 144 weeks ]
  3. Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 144 weeks ]
  4. Change from Baseline in Quantity of Microdystrophin Protein Expression [ Time Frame: Baseline, Week 12 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Inheritance of DMD follows an X-linked recessive pattern. Birth prevalence has been estimated at 1 in 3500 to 5000 live male births.
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
  • Indication of symptomatic muscular dystrophy by protocol-specified criteria.
  • Ability to cooperate with motor assessment testing.
  • Stable dose equivalent of oral corticosteroids for at least 12 weeks.

Exclusion Criteria:

  • Impaired cardiovascular function on ECHO.
  • Prior or ongoing medical condition on physical examination, ECG, or laboratory findings that could adversely affect subject safety, compromise completion of follow-up, or impair assessment of study results.
  • Exposure to another investigational drug or exon skipping medication within months.
  • Exposure to an investigational or commercial gene therapy product.
  • Abnormal liver or renal function by protocol-specified criteria

Other inclusion/exclusion criteria apply.


Information from the National Library of Medicine

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): NCT03769116


Contacts
Contact: Medical Information +1-888-727-3782 clinicaltrials@sarepta.com

Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Study Director: Medical Director Sarepta Therapeutics, Inc.

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT03769116     History of Changes
Other Study ID Numbers: SRP-9001-102
First Posted: December 7, 2018    Key Record Dates
Last Update Posted: December 7, 2018
Last Verified: December 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sarepta Therapeutics:
Duchenne Muscular Dystrophy
Gene-Delivery
DMD
Ambulatory
Pediatric
North Star Ambulatory Assessment (NSAA)

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked