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Clinical Trials in the UK / NCT05524883
Active, not recruiting Phase 1/2

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

NCT05524883 · tracked via the Priya Life Science UK tracker
Sponsor
Dyne Therapeutics
Phase
Phase 1/2
Started
2022-08-12
Last updated
2026-08-11

Condition(s) studied

Duchenne Muscular Dystrophy (DMD)

Investigational drug(s) / intervention(s)

DYNE-251Placebo

DYNE-251: Administered by IV infusion

Placebo: Administered by IV infusion

Study 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 (LTE) period (288 weeks).

Eligibility

Sex
MALE
Min age
4 Years
Max age
16 Years
Healthy volunteers
No
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 enrollment. * 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.

Primary outcome measure(s)

Trial sites (30)

FacilityCityRegionStatus
University of California San Diego La Jolla California
UCLA University California of Los Angeles Los Angeles California
Children's Hospital Colorado Aurora Colorado
Rare Disease Research, LLC Atlanta Georgia
UMass Memorial Medical Center Worcester Massachusetts
Nationwide Children's Hospital Columbus Ohio
Shriners Hospitals for Children Portland Portland Oregon
Children's Hospital of Philadelphia Philadelphia Pennsylvania
UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
University of Utah - PPDS Salt Lake City Utah
Virginia Commonwealth University Richmond Virginia
Children's Hospital at Westmead Westmead New South Wales
Murdoch Children's Research Institute Parkville Victoria
UZ Gent Ghent Belgium
UZ Leuven Leuven Belgium
CHR Citadelle Liège Belgium
London Health Sciences Centre London Ontario
Children's Hospital of Eastern Ontario Ottawa Ontario
CHI [Children's Health Ireland] at Temple Street Children's University Hospital Dublin Ireland
Fondazione Policlinico Universitario A Gemelli Rome Lazio
Fondazione Serena Onlus - Centro Clinico NeMO Milan Lombardy
Ospedale San Raffaele S.r.l. - PPDS Milan Lombardy
Samsung Medical Center Seoul Teugbyeolsi
Hospital Universitario Vall d'Hebron - PPDS Barcelona Spain
Hospital Sant Joan de Déu Universidad de Barcelona Barcelona Spain
Alder Hey Children's Hospital Liverpool Merseyside
Royal Victoria Infirmary Newcastle upon Tyne Northumberland
Leeds Teaching Hospitals NHS Trust Leeds West Yorkshire
Bristol Childrens Hospital Bristol United Kingdom
Great Ormond Street Hospital London United Kingdom

More Dyne Therapeutics trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05524883 on ClinicalTrials.gov ↗ ← All trials in the UK