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Phase I/II Study of SRP-4053 in DMD Patients

NCT02310906 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 39

Last updated 2020-10-19

Study results available
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Summary

This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.

Conditions

Interventions

DRUG

Placebo

SRP-4053 placebo-matching solution for IV infusion.

DRUG

SRP-4053

SRP-4053 (golodirsen) solution for IV infusion.

Sponsors & Collaborators

  • Institut de Myologie, France

    collaborator OTHER

  • Consultants for Research in Imaging and Spectroscopy

    collaborator OTHER

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    collaborator OTHER

  • Catholic University of the Sacred Heart

    collaborator OTHER

  • Royal Holloway University

    collaborator OTHER

  • SYSNAV

    collaborator INDUSTRY

  • University College, London

    collaborator OTHER

  • University of Newcastle Upon-Tyne

    collaborator OTHER

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
15 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-01-13
Primary Completion
2019-03-25
Completion
2019-03-25
FDA Drug
Yes

Countries

  • United States
  • France
  • Italy
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02310906 on ClinicalTrials.gov