Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Avidity Biosciences

Status and phase: Enrolling; Phase 3

Conditions: Steinert; Myotonic Dystrophy Type 1 (DM1); Myotonia; Myotonic Dystrophy 1; DM1; Myotonic Disorders; Myotonic Muscular Dystrophy; Dystrophy Myotonic; Steinert Disease; Myotonic Dystrophy

Treatments: Drug: Placebo; Drug: AOC 1001 (del-desiran)

Study type: Interventional

Funder types: Industry

Identifiers

NCT06411288

AOC 1001-CS3

Details and patient eligibility

About

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

Full 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.

Enrollment: 150 estimated patients

Sex: All

Ages: 16 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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150 participants in 2 patient groups, including a placebo group

Del-desiran

Experimental group

Description:

Del-desiran (AOC 1001) will be administered seven times

Treatment:

Drug: AOC 1001 (del-desiran)

Placebo

Placebo Comparator group

Description:

Saline will be administered seven times

Treatment:

Drug: Placebo