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Trial record 1 of 2 for:    DMD EXON 45 (SRP-4045)
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Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics
ClinicalTrials.gov Identifier:
NCT02530905
First received: August 10, 2015
Last updated: June 14, 2017
Last verified: June 2017
  Purpose
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.

Condition Intervention Phase
Duchenne Muscular Dystrophy Drug: SRP-4045 Drug: Placebo Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Sarepta Therapeutics:

Primary Outcome Measures:
  • Incidence of adverse events [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]

Secondary Outcome Measures:
  • Drug concentration in plasma [ Time Frame: Approximately 12 Weeks (double-blind dose titration) ]

Estimated Enrollment: 12
Study Start Date: October 2015
Estimated Study Completion Date: April 2018
Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SRP-4045 (double-blind dose titration)
Approximately 8 genotypically confirmed DMD patients amenable to exon 45 skipping
Drug: SRP-4045
Placebo Comparator: Placebo (double-blind dose titration)
Approximately 4 genotypically confirmed DMD patients amenable to exon 45 skipping.
Drug: Placebo
Experimental: SRP-4045 (open label)
Approximately 12 DMD patients who completed the double-blind dose titration part of the study.
Drug: SRP-4045

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.

  Eligibility

Ages Eligible for Study:   7 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02530905

Locations
United States, California
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Study Director: Genevieve Laforet, MD, PhD Sarepta Therapeutics
Study Chair: Edward Kaye, MD Sarepta Therapeutics
  More Information

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT02530905     History of Changes
Other Study ID Numbers: 4045-101
Study First Received: August 10, 2015
Last Updated: June 14, 2017

Keywords provided by Sarepta Therapeutics:
Duchenne muscular dystrophy
Exon Skipping

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

ClinicalTrials.gov processed this record on June 23, 2017