DYNE-251 Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy: DELIVER Clinical Trial
Study Overview
- Age
- 4–16 years
- Phase
- Phase 1/2
- Sponsor
- Dyne Therapeutics
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2022-08-12
- Primary Completion (Estimated)
- 2029-11
- Study Completion (Estimated)
- 2029-11
- Enrollment (Estimated)
- 86
- Countries
- United StatesAustraliaBelgiumCanadaIrelandItalySouth KoreaSpainUnited Kingdom
Study Requirements and Criteria
Steroid Use
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).
Inclusion Criteria
- Age 4 to 16 years inclusive, at the time of informed consent/assent.
- 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.
- 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.
Contacts and Locations
Contacts and Locations
Contact
- Name: Dyne Clinical Trials
- Phone Number: +1-781-317-1919
- Email: [email protected]
Locations
This study has 30 locations
United States
California Locations
La Jolla, California, United States, 92037
University of California San Diego
Los Angeles, California, United States, 90095
UCLA University California of Los Angeles
Colorado Locations
Aurora, Colorado, United States, 80045
Children's Hospital Colorado
Georgia Locations
Atlanta, Georgia, United States, 30329
Rare Disease Research, LLC
Massachusetts Locations
Worcester, Massachusetts, United States, 01655
UMass Memorial Medical Center
Ohio Locations
Columbus, Ohio, United States, 43205
Nationwide Children's Hospital
Oregon Locations
Portland, Oregon, United States, 97239
Shriners Hospitals for Children Portland
Pennsylvania Locations
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Philadelphia
Pittsburgh, Pennsylvania, United States, 15224-1334
UPMC Children's Hospital of Pittsburgh
Utah Locations
Salt Lake City, Utah, United States, 08412
University of Utah - PPDS
Virginia Locations
Richmond, Virginia, United States, 23219
Virginia Commonwealth University
Australia
New South Wales Locations
Westmead, New South Wales, Australia, 02145
Children's Hospital at Westmead
Victoria Locations
Parkville, Victoria, Australia, 3052
Murdoch Children's Research Institute
Belgium
Ghent, Belgium, 9000
UZ Gent
Leuven, Belgium, 3000
UZ Leuven
Liège, Belgium, 4000
CHR Citadelle
Canada
Ontario Locations
London, Ontario, Canada, N6A 5W9
London Health Sciences Centre
Ottawa, Ontario, Canada, ON K1H 8L1
Children's Hospital of Eastern Ontario
Ireland
Dublin, Ireland, D01 XD99
CHI [Children's Health Ireland] at Temple Street Children's University Hospital
Italy
Lazio Locations
Rome, Lazio, Italy, 00168
Fondazione Policlinico Universitario A Gemelli
Lombardy Locations
Milan, Lombardy, Italy, 20162
Fondazione Serena Onlus - Centro Clinico NeMO
Milan, Lombardy, Italy, 20312
Ospedale San Raffaele S.r.l. - PPDS
South Korea
Teugbyeolsi Locations
Seoul, Teugbyeolsi, South Korea, 6351
Samsung Medical Center
Spain
Barcelona, Spain, 8025
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Spain, 8950
Hospital Sant Joan de Déu Universidad de Barcelona
United Kingdom
Merseyside Locations
Liverpool, Merseyside, United Kingdom, L12 2AP
Alder Hey Children's Hospital
Northumberland Locations
Newcastle upon Tyne, Northumberland, United Kingdom, NE1 4LP
Royal Victoria Infirmary
West Yorkshire Locations
Leeds, West Yorkshire, United Kingdom, LS1 3EX
Leeds Teaching Hospitals NHS Trust
Bristol, United Kingdom, BS2 8BJ
Bristol Childrens Hospital
London, United Kingdom, WC1N 3JH
Great Ormond Street Hospital
Clinical Trial Registry
NCT ID
NCT05524883This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.