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Antisense Oligonucleotide

Del-desiran for Myotonic Dystrophy (HARBOR Trial)

Phase 3
Recruiting
Research Sponsored by Avidity Biosciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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
Must not have
Unwilling or unable to comply with contraceptive requirements
Diabetes that is not adequately controlled
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through week 30
Awards & highlights
Pivotal Trial

Summary

This trial aims to test the effectiveness and safety of a new drug called del-desiran in treating Myotonic Dystrophy Type 1. It will be a global study involving a large number of

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects aren't listed here, typical ones may include reactions at the infusion site, potential allergic responses to the medication components or general side effects like headaches or nausea.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with DM1 and my genetic test shows CTG repeat ≥ 100.
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I can walk by myself for at least 10 meters, even with supports like braces.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I cannot or will not follow the birth control requirements.
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My diabetes is not well-controlled.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through week 30
This trial's timeline: 3 weeks for screening, Varies for treatment, and through week 30 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Hand function
Secondary study objectives
Hand grip strength
Quantitative Muscle Testing composite score

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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

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Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor
7 Previous Clinical Trials
796 Total Patients Enrolled
2 Trials studying Myotonic Dystrophy
75 Patients Enrolled for Myotonic Dystrophy
~100 spots leftby Oct 2026
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