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Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

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Brief 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).

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase 1/Phase 2

Eligibility

Gender
Male
Healthy Volunteers
No
Minimum Age
4 Years
Maximum Age
16 Years

Inclusion Criteria:

  • Age 4 to 16 years inclusive, at the time of informed consent/assent.
  • Male with a confirmed diagnosis of DMD and with a 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 Periods 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).

Exclusion Criteria:

  • 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.

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Study Stats
Protocol No.
22-001705
Category
Genetic and Rare Diseases
Musculoskeletal Disorders
Contact
UMMULWARA QASIM
Location
  • UCLA Westwood
For Providers
NCT No.
NCT05524883
For detailed technical eligibility, visit ClinicalTrials.gov.