Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
NCT ID: NCT05524883
Last Updated: 2025-08-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
86 participants
INTERVENTIONAL
2022-08-12
2029-11-30
Brief Summary
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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 (192 weeks).
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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Placebo-Controlled MAD Period - DYNE-251
DYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.
DYNE-251
Administered by IV infusion
Placebo-Controlled MAD Period - Placebo
Placebo will be administered Q4W or Q8W over 24 weeks.
Placebo
Administered by IV infusion
Open-Label and Long-Term Extension Period - DYNE-251
DYNE-251 will be administered Q4W or Q8W for up to 192 weeks after participants complete the Placebo-Controlled MAD Period of the study.
DYNE-251
Administered by IV infusion
Interventions
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DYNE-251
Administered by IV infusion
Placebo
Administered by IV infusion
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
4 Years
16 Years
MALE
No
Sponsors
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Dyne Therapeutics
INDUSTRY
Responsible Party
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Locations
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University of California San Diego
La Jolla, California, United States
UCLA University California of Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
UMass Memorial Medical Center
Worcester, Massachusetts, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Shriners Hospitals for Children Portland
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah - PPDS
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Children's Hospital at Westmead
Westmead, New South Wales, Australia
Murdoch Children's Research Institute
Parkville, Victoria, Australia
UZ Gent
Ghent, , Belgium
UZ Leuven
Leuven, , Belgium
CHR Citadelle
Liège, , Belgium
London Health Sciences Centre
London, Ontario, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
CHI [Children's Health Ireland] at Temple Street Children's University Hospital
Dublin, , Ireland
Fondazione Policlinico Universitario A Gemelli
Rome, Lazio, Italy
Fondazione Serena Onlus - Centro Clinico NeMO
Milan, Lombardy, Italy
Ospedale San Raffaele S.r.l. - PPDS
Milan, Lombardy, Italy
Samsung Medical Center
Seoul, Teugbyeolsi, South Korea
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, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, Northumberland, United Kingdom
Leeds Teaching Hospitals NHS Trust
Leeds, West Yorkshire, United Kingdom
Bristol Childrens Hospital
Bristol, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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Other Identifiers
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2023-510351-31-00
Identifier Type: CTIS
Identifier Source: secondary_id
ITA 362546 (model PA-000798)
Identifier Type: OTHER
Identifier Source: secondary_id
DYNE251-DMD-201
Identifier Type: -
Identifier Source: org_study_id
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