A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD (FORTITUDE-3)

Avidity Biosciences

Status and phase: Enrolling; Phase 3

Conditions: Facioscapulohumeral Dystrophy; Landouzy-Dejerine Muscular Dystrophy; FSHD - Facioscapulohumeral Muscular Dystrophy; Facio-Scapulo-Humeral Dystrophy; Facioscapulohumeral Muscular Dystrophy 1; Fascioscapulohumeral Muscular Dystrophy; FSHD1; Fascioscapulohumeral Muscular Dystrophy Type 1; FSH Muscular Dystrophy; Landouzy Dejerine Dystrophy; Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1); Facioscapulohumeral Muscular Dystrophy 2; Fascioscapulohumeral Muscular Dystrophy Type 2; FSHD; FSHD2; Facioscapulohumeral Muscular Dystrophy; Landouzy-Dejerine Syndrome

Treatments: Drug: Placebo; Drug: AOC-1020

Study type: Interventional

Funder types: Industry

Identifiers

NCT07038200

AOC 1020-CS3

Details and patient eligibility

About

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Full description

The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose).

Participants will be randomized to receive an intravenous infusion of either delbrax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.

After completion of the Week 78 visit, eligible participants will have the option to enroll in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 12 weeks for safety.

An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Enrollment: 200 estimated patients

Sex: All

Ages: 16 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical and genetic diagnosis of FSHD1 or FSHD2
• Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
• Adequate muscle strength based on QMT composite score

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
• Blood Pressure > 140/90 mmHg at Screening
• Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
• Treatment with an oligonucleotide within 9 months of Screening

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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200 participants in 2 patient groups, including a placebo group

Del-brax

Experimental group

Description:

Del-brax (AOC 1020) will be administered 13 times

Treatment:

Drug: AOC-1020

Placebo

Placebo Comparator group

Description:

Saline will be administered 13 times

Treatment:

Drug: Placebo