Del-desiran for Myotonic Dystrophy
(HARBOR Trial)

Recruiting in Palo Alto (17 mi)

+41 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Avidity Biosciences, Inc.

Must not be taking: Anti-myotonic medications

Disqualifiers: Pregnancy, Uncontrolled diabetes, Heart failure, others

Pivotal Trial (Near Approval)

Prior Safety Data

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

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Trial 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 (Antisense Oligonucleotide)

Trial OverviewThe 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

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Del-desiranExperimental Treatment1 Intervention

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

Group II: PlaceboPlacebo Group1 Intervention

Saline will be administered seven times