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

A

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: AOC-1020
Drug: Placebo

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 consists of a Screening Period of up to 6 weeks and 72-week Treatment Period. The anticipated duration is approximately 78 weeks.

Participants will be randomized to receive an intravenous infusion of either del-brax 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 into an open label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for a period of 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

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

Trial contacts and locations

13

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Central trial contact

Avidity Biosciences, Inc.

Data sourced from clinicaltrials.gov

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