Global Study of Del-desiran for the Treatment of DM1
HARBOR
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
1 other identifier
interventional
159
10 countries
34
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started May 2024
34 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 8, 2024
CompletedFirst Posted
Study publicly available on registry
May 13, 2024
CompletedStudy Start
First participant enrolled
May 30, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2026
January 22, 2026
January 1, 2026
2.2 years
May 8, 2024
January 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Hand function
video Hand Opening Time (vHOT)
Through Week 54
Secondary Outcomes (4)
Hand grip strength
Through Week 54
Quantitative Muscle Testing composite score
Through Week 54
Myotonic Dystrophy Type 1 activity and participation scale c
Through Week 54
10-Meter Walk/Run Test
Through Week 54
Study Arms (2)
Del-desiran
EXPERIMENTALDel-desiran (AOC 1001) will be administered seven times
Placebo
PLACEBO COMPARATORSaline will be administered seven times
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (34)
Stanford University
Stanford, California, 94305, United States
University of Colorado
Denver, Colorado, 80045, United States
University of Florida
Gainesville, Florida, 32608, United States
University Research Center of South Florida
Tampa, Florida, 33612, United States
Indiana University (IU)
Indianapolis, Indiana, 46202, United States
Kansas University Medical Center
Kansas City, Kansas, 66205, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Duke University Medical Center
Durham, North Carolina, 27708, United States
Wake Forest
Winston-Salem, North Carolina, 27157, United States
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, 45219, United States
Ohio State University
Columbus, Ohio, 43221, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Houston Methodist Neurological Institute
Houston, Texas, 77030, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
University of Washington
Seattle, Washington, 98104, United States
The Ottawa Hospital
Ottawa, Ontario, K1Y 4E9, Canada
Montreal Neurological Institute
Montreal, Quebec, H3A 2B4, Canada
Aarhus University Hospital
Aarhus N, 8200, Denmark
Rigshospitalet
Copenhagen, 2100, Denmark
AP-HP Hopital Pitie-Salpetriere
Paris, 75013, France
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, 80336, Germany
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, 20162, Italy
Aomori Hospital
Aomori, Aomori, 038-1331, Japan
National Hospital Organization Osaka Toneyama Medical Center
Osaka, 560-8552, Japan
Osaka University Hospital
Osaka, 565-0871, Japan
National Center of Neurology and Psychiatry
Tokyo, 187-8551, Japan
Maastricht University Medical Center
Maastricht, 6229 HX, Netherlands
Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA, Netherlands
Hospital Universitario Donostia
Donostia / San Sebastian, 20014, Spain
University College London Hospital
London, NW1 2BU, United Kingdom
St. Georges University Hospitals NHS Foundation Trust
London, SW17 0QT, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 8, 2024
First Posted
May 13, 2024
Study Start
May 30, 2024
Primary Completion (Estimated)
August 1, 2026
Study Completion (Estimated)
September 1, 2026
Last Updated
January 22, 2026
Record last verified: 2026-01