An open-label extension study for patients with Duchenne muscular dystrophy who participated in studies of SRP-5051 | DuchenneXchange

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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: January 21, 2019

study details

start date: December 19, 2018

estimated completion: November 1, 2020

phase of development: Phase 1/Phase 2

size / enrollment: 30

primary outcomes:

  • Number of participants with adverse events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
    An AE is any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug, whether or not considered related to the study drug.

secondary outcomes:

  • Maximum plasma concentration (Cmax) of SRP-5051 [ Time Frame: End of infusion ]
    Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
  • Area under the plasma concentration versus time curve (AUC) of SRP-5051 [ Time Frame: Pre-dose, end of infusion, 6 hours post-dose ]
    Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug 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:
• Has completed Study 5051-101 (NCT03375255) and continues to meet the Safety Eligibility Criteria of Study 5051-101 (NCT03375255).

exclusion criteria:
• Initiation or change of dosing (except for modifications to accommodate changes in weight) since entering Study 5051-101 (NCT03375255) 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.

locations: United States