- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06411288
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
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Contacts and Locations
Study Contact
- Name: Avidity Biosciences, Inc.
- Phone Number: 858-771-7038
- Email: medinfo@aviditybio.com
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Key Inclusion Criteria:
- 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 Exclusion Criteria:
- 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 Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Del-desiran
Del-desiran (AOC 1001) will be administered seven times
|
Del-desiran will be administered by intravenous (IV) infusion.
|
Placebo Comparator: Placebo
Saline will be administered seven times
|
Placebo will be administered by intravenous (IV) infusion.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Hand function
Time Frame: Through Week 30
|
video Hand Opening Time (vHOT)
|
Through Week 30
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Hand grip strength
Time Frame: Through Week 30
|
by dynamometer
|
Through Week 30
|
Quantitative Muscle Testing composite score
Time Frame: Through Week 30
|
by dynamometer
|
Through Week 30
|
Myotonic Dystrophy Type 1 activity and participation scale c
Time Frame: Through Week 30
|
Through Week 30
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Muscular Disorders, Atrophic
- Heredodegenerative Disorders, Nervous System
- Muscular Dystrophies
- Myotonic Dystrophy
- Myotonia
- Myotonic Disorders
Other Study ID Numbers
- AOC 1001-CS3
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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