AOC 1001-CS3 HARBOR | Clinical Research Studies Listing

Recruiting

18 years - 65 years

All

Phase 3

1 Location

Brief description of study

This is a phase 3 trial of AOC 1001, an antibody-oligonucleotide conjugate. Protocol includes participants ages 16-65 with a clinical and genetic diagnosis of DM1 with DMPK CTG repeat length greater than or equal to 100; Penn site will enroll ages 18 to 65 years only. Participants will be randomized (1:1) to receive an IV infusion of AOC 1001 or placebo every 8 weeks for 7 doses. Participants will undergo physical/neuro exams, ECGs, blood/urine tests, function/strength tests, and questionnaires.

Detailed description of study

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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

Conditions: Medical Research
Age: 18 years - 65 years
Gender: All

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

Updated on 18 Mar 2025. Study ID: 24-1096

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