Del-desiran for Myotonic Dystrophy

(HARBOR Trial)

Recruiting at 41 trial locations

Overseen By Avidity Biosciences, Inc.

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Avidity Biosciences, Inc.

Must not be taking: Anti-myotonic medications

Disqualifiers: Pregnancy, Uncontrolled diabetes, Heart failure, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 7

Duration: 54 weeks

150 Participants Needed

Trial Summary

What is the purpose of this trial?

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

Will I have to stop taking my current medications?

The trial requires that you stop taking any anti-myotonic medications at least 14 days before starting the study or longer, depending on how long the medication stays in your body.

Eligibility Criteria

This trial is for individuals who can walk independently (with orthoses and ankle braces allowed) at least 10 meters and have a clinical and genetic diagnosis of Myotonic Dystrophy Type 1 with CTG repeat ≥ 100. It's not specified who cannot join.

Inclusion Criteria

I can walk by myself for at least 10 meters, even with supports like braces.

I have been diagnosed with DM1 and my genetic test shows CTG repeat ≥ 100.

Exclusion Criteria

I cannot or will not follow the birth control requirements.

My diabetes is not well-controlled.

Body Mass Index > 35 kg/m2 at Screening

Timeline

Screening

Participants are screened for eligibility to participate in the trial

up to 6 weeks

Treatment

Participants receive an intravenous infusion of either del-desiran or placebo every 8 weeks for a total of 7 doses

54 weeks

7 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 weeks

Open-label extension (optional)

Eligible participants may opt into continuation of treatment long-term, pending regulatory approval

Treatment Details

Interventions

• Del-desiran

Trial Overview The study tests the efficacy and safety of an intravenous drug called del-desiran, compared to a placebo in treating Myotonic Dystrophy Type 1. This phase 3 trial is randomized, meaning participants are randomly assigned to groups, and double-blind, so neither researchers nor participants know who gets the real treatment.

Participant Groups

Group I: Del-desiran Experimental Treatment 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

Saline will be administered seven times

Who Is Running the Clinical Trial?

Avidity Biosciences, Inc.

Lead Sponsor

Trials

8

Recruited

960+