Global Study of Del-desiran for the Treatment of DM1
- Conditions
- Myotonic Dystrophy 1Myotonic DisordersDM1Myotonic DystrophySteinertDystrophy MyotonicSteinert DiseaseMyotonic Muscular DystrophyMyotoniaMyotonic Dystrophy Type 1 (DM1)
- Interventions
- Drug: AOC 1001 (del-desiran)Drug: Placebo
- Registration Number
- NCT06411288
- Lead Sponsor
- Avidity Biosciences, Inc.
- Brief Summary
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
- Detailed Description
The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.
Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.
After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
- Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
- Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Key
- Breastfeeding, pregnancy, or intent to become pregnant during the study
- Unwilling or unable to comply with contraceptive requirements
- Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- Diabetes that is not adequately controlled
- History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
- Body Mass Index > 35 kg/m2 at Screening
- Recently treated with an investigational drug or biological agent
- Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.
Note: Additional protocol defined Inclusion and Exclusion criteria apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Del-desiran AOC 1001 (del-desiran) Del-desiran (AOC 1001) will be administered seven times Placebo Placebo Saline will be administered seven times
- Primary Outcome Measures
Name Time Method Hand function Through Week 30 video Hand Opening Time (vHOT)
- Secondary Outcome Measures
Name Time Method Hand grip strength Through Week 30 by dynamometer
Myotonic Dystrophy Type 1 activity and participation scale c Through Week 30 Quantitative Muscle Testing composite score Through Week 30 by dynamometer
Trial Locations
- Locations (42)
University of California, Los Angeles (UCLA)
🇺🇸Los Angeles, California, United States
University of California, San Diego (UCSD)
🇺🇸San Diego, California, United States
Stanford University
🇺🇸Stanford, California, United States
University of Colorado
🇺🇸Denver, Colorado, United States
University of Florida
🇺🇸Gainesville, Florida, United States
University Research Center of South Florida
🇺🇸Tampa, Florida, United States
Indiana University (IU)
🇺🇸Indianapolis, Indiana, United States
Kansas University Medical Center
🇺🇸Kansas City, Kansas, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Wake Forest
🇺🇸Winston-Salem, North Carolina, United States
University of Cincinnati Gardner Neuroscience Institute
🇺🇸Cincinnati, Ohio, United States
Ohio State University
🇺🇸Columbus, Ohio, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Houston Methodist Neurological Institute
🇺🇸Houston, Texas, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
University of Washington
🇺🇸Seattle, Washington, United States
The Ottawa Hospital
🇨🇦Ottawa, Ontario, Canada
CSSS de Chicoutimi
🇨🇦Chicoutimi, Quebec, Canada
Montreal Neurological Institute
🇨🇦Montreal, Quebec, Canada
Aarhus University Hospital
🇩🇰Aarhus N, Denmark
Rigshospitalet
🇩🇰Kobenhavn, Denmark
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
🇫🇷Marseille, France
AP-HP Hopital Pitie-Salpetriere
🇫🇷Paris, France
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
🇩🇪Munich, Germany
Beaumont Hospital
🇮🇪Dublin, Ireland
Fondazione Serena Onlus - Centro Clinico NeMO Milano
🇮🇹Milano, Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
🇮🇹Rome, Italy
Aomori Hospital
🇯🇵Aomori-shi, Aomori, Japan
National Hospital Organization Osaka Toneyama Medical Center
🇯🇵Osaka, Japan
Osaka University Hospital
🇯🇵Osaka, Japan
National Center of Neurology and Psychiatry
🇯🇵Toyko, Japan
Maastricht University Medical Center
🇳🇱Maastricht, Netherlands
Stichting Radboud Universitair Medisch Centrum
🇳🇱Nijmegen, Netherlands
Hospital Universitario Donostia
🇪🇸San Sebastian, Spain
University College London Hospital
🇬🇧London, United Kingdom
St. Georges University Hospitals NHS Foundation Trust
🇬🇧London, United Kingdom
Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom
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