Summary

Eligibility
for males ages 4-16 (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
completion around

Description

Summary

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).

Official Title

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Keywords

Duchenne Muscular Dystrophy (DMD), Muscular Dystrophies, Duchenne Muscular Dystrophy, DYNE-251

Eligibility

You can join if…

Open to males ages 4-16

  • Age 4 to 16 years inclusive, at the time of informed consent/assent.
  • Male with a confirmed DMD mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
  • Upper extremity muscle group that is amenable to muscle biopsy.
  • Brooke Upper Extremity Scale score of 1 or 2.
  • Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrolment.
  • Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration, with the expectation of maintaining a stable dose during the Placebo-controlled and Open-label Period of the study (unless dose adjustment is required by weight change).
  • Left ventricular ejection fraction of ≥50% by echocardiogram or ≥55% by cardiac magnetic resonance imaging (MRI).

You CAN'T join if...

  • Uncontrolled clinical symptoms and signs of congestive heart failure (CHF).
  • Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment.
  • History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study.
  • Requirement of daytime ventilator assistance.
  • Percent predicted FVC <40 % (applies only for participants who are age ≥7 years).
  • Receipt of eteplirsen, or alternative exon-skipping/dystrophin-modifying therapy, within 12 weeks of randomization.
  • Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration.
  • Receipt of gene therapy at any time.

    Other inclusion and exclusion criteria may apply.

Locations

  • University of California San Diego accepting new patients
    La Jolla California 92037 United States
  • UCLA accepting new patients
    Los Angeles California 90095 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Dyne Therapeutics
ID
NCT05524883
Phase
Phase 1/2 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 48 study participants
Last Updated