Global Study of Del-desiran for the Treatment of DM1

Myotonic Dystrophy 1 Clinical Trial

— HARBOR  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06411288
• Other study ID #: AOC 1001-CS3
• Status: Recruiting
• Phase: Phase 3
• Start date: May 30, 2024
• Est. completion date: April 2027

Study information

• Verified date: June 2024
• Source: Avidity Biosciences, Inc.
• Contact: Avidity Biosciences, Inc.
• Phone: 858-771-7038
• Email: medinfo[@]aviditybio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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 information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: April 2027
• Est. primary completion date: October 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• DM1
• Muscular Dystrophies
• Myotonia
• Myotonic Disorders
• Myotonic Dystrophy
• Myotonic Dystrophy 1
• Myotonic Dystrophy Type 1 (DM1)
• Myotonic Muscular Dystrophy
• Steinert Disease

Intervention

Drug:
• AOC 1001 (del-desiran)
Del-desiran will be administered by intravenous (IV) infusion.
• Placebo
Placebo will be administered by intravenous (IV) infusion.

Locations

Country	Name	City	State
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of Rochester Medical Center	Rochester	New York

Sponsors (1)

Lead Sponsor	Collaborator
Avidity Biosciences, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Hand function	video Hand Opening Time (vHOT)	Through Week 30
Secondary	Hand grip strength	by dynamometer	Through Week 30
Secondary	Quantitative Muscle Testing composite score	by dynamometer	Through Week 30
Secondary	Myotonic Dystrophy Type 1 activity and participation scale c		Through Week 30