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

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ClinicalTrials.gov Identifier: NCT03675126
Recruitment Status : Recruiting
First Posted : September 18, 2018
Last Update Posted : August 1, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE August 6, 2018
First Posted Date  ICMJE September 18, 2018
Last Update Posted Date August 1, 2019
Actual Study Start Date  ICMJE December 19, 2018
Estimated Primary Completion Date July 31, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 30, 2019)
Incidence of Adverse Events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 14, 2018)
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.
Change History Complete list of historical versions of study NCT03675126 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 30, 2019)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: September 14, 2018)
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Official Title  ICMJE An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE Drug: SRP-5051
SRP-5051 administered as an IV infusion.
Study Arms  ICMJE Experimental: SRP-5051
Patients will receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 studies.
Intervention: Drug: SRP-5051
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 30, 2019)
54
Original Estimated Enrollment  ICMJE
 (submitted: September 14, 2018)
30
Estimated Study Completion Date  ICMJE July 31, 2024
Estimated Primary Completion Date July 31, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

• 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.

Other inclusion/exclusion criteria apply.

* The dose of steroids must remain constant except for modifications to accommodate changes in weight.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 7 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical information +1 888 727 3782 clinicaltrials@sarepta.com
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03675126
Other Study ID Numbers  ICMJE 5051-102
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sarepta Therapeutics, Inc.
Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP