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Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

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ClinicalTrials.gov Identifier: NCT02530905
Recruitment Status : Completed
First Posted : August 21, 2015
Last Update Posted : November 26, 2018
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Tracking Information
First Submitted Date  ICMJE August 10, 2015
First Posted Date  ICMJE August 21, 2015
Last Update Posted Date November 26, 2018
Actual Study Start Date  ICMJE October 14, 2015
Actual Primary Completion Date October 3, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 19, 2015)
Incidence of adverse events [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02530905 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 19, 2015)
Drug concentration in plasma [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping
Brief Summary This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping.
Detailed Description

This is a randomized, placebo-controlled dose-titration study to assess safety, tolerability, and pharmacokinetics of 4 dose levels of SRP-4045 in genotypically confirmed advanced-stage DMD patients with deletions amenable to exon 45 skipping.

After completion of the dose-titration portion of the study and SRP-4045 is determined to be safe, all patients will be evaluated on open-label SRP-4045 for the duration of the study.

Safety, including adverse event monitoring, routine laboratory assessments, and cardiac testing will be monitored through the duration of the dose-titration and open-label portions of the study.

Clinical efficacy will be assessed at regularly scheduled study visits via quality of life questionnaires and tests of pulmonary and upper extremity function through the duration of the dose-titration and open-label portions of the trial.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: SRP-4045
  • Drug: Placebo
Study Arms  ICMJE
  • Experimental: SRP-4045 (double-blind dose titration)
    Approximately 8 genotypically confirmed DMD patients amenable to exon 45 skipping
    Intervention: Drug: SRP-4045
  • Placebo Comparator: Placebo (double-blind dose titration)
    Approximately 4 genotypically confirmed DMD patients amenable to exon 45 skipping.
    Intervention: Drug: Placebo
  • Experimental: SRP-4045 (open label)
    Approximately 12 DMD patients who completed the double-blind dose titration part of the study.
    Intervention: Drug: SRP-4045
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 Completed
Estimated Enrollment  ICMJE
 (submitted: August 19, 2015)
12
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 3, 2018
Actual Primary Completion Date October 3, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Genotypically confirmed DMD (amenable to exon 45 skipping).
  • Stable cardiac and pulmonary function.
  • Limited or no ambulation.
  • On a stable dose of oral corticosteroids for at least 24 weeks OR has not received corticosteroids for at least 24 weeks.

Exclusion Criteria:

  • Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or PRO045.
  • Other experimental treatment in the past 12 weeks.
  • If on cardiac medication, must be on a stable dose for the past 12 weeks.
  • Major surgery within the past 3 months.

Other inclusion/exclusion criteria apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 7 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02530905
Other Study ID Numbers  ICMJE 4045-101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sarepta Therapeutics
Study Sponsor  ICMJE Sarepta Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics
Verification Date November 2018

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