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An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy who Participated in Studies of SRP-5051

key information

study id #: NCT03675126

condition: Muscular Dystrophy, Duchenne

status: recruiting

purpose:

The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

intervention: SRP-5051

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03675126

last updated: August 14, 2019

study details

start date: December 19, 2018

estimated completion: July 31, 2024

phase of development: Phase 1/Phase 2

size / enrollment: 54

primary outcomes:

  • Incidence of Adverse Events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]

secondary outcomes:

  • Pharmacokinetic (PK) Plasma Concentration of SRP-5051 [ Time Frame: End of infusion ]
  • Number of participants with clinically relevant abnormalities, as assessed by vital sign measurements, physical examination findings, clinical laboratory tests and electrocardiograms (ECGs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
    *A clinically relevant abnormality is an abnormality confirmed by repeat testing that is changed sufficiently from screening/baseline so that, in the judgment of the Investigator, a change in management is warranted.

inclusion criteria:

• Eligible Sexes:

• Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.

exclusion criteria:
• Initiation or change of dosing (except for modifications to accommodate changes in weight) since completing a study administering SRP-5051 and while participating in this study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids*.
• Requires antiarrhythmic and/or diuretic therapy for heart failure.
• Use of any herbal medication/supplement containing aristolochic acid.
• Treatment with any experimental therapy since entering original study or any experimental gene therapy for the treatment of DMD at any time.
• Participation in an interventional clinical trial since completing original study.

study contacts

sponsor: Sarepta Therapeutics

contacts: Medical Information, +1 888 727 3782, Clinicaltrials@sarepta.com

investigators: Medical Director; Sarepta Therapeutics, Inc.

trial center locations: United States, Canada