A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
Purpose
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)
Conditions
- Facioscapulohumeral Muscular Dystrophy
- FSHD
- FSHD - Facioscapulohumeral Muscular Dystrophy
- FSHD1
- FSHD2
- Fascioscapulohumeral Muscular Dystrophy
- Fascioscapulohumeral Muscular Dystrophy Type 1
- Fascioscapulohumeral Muscular Dystrophy Type 2
- Facioscapulohumeral Muscular Dystrophy 1
- Facioscapulohumeral Dystrophy
- Facio-Scapulo-Humeral Dystrophy
- Facioscapulohumeral Muscular Dystrophy 2
- Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)
- FSH Muscular Dystrophy
- Landouzy Dejerine Dystrophy
- Landouzy-Dejerine Muscular Dystrophy
- Landouzy-Dejerine Syndrome
Eligibility
- Eligible Ages
- Between 16 Years and 70 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental del-brax |
Del-brax (AOC 1020) will be administered 13 times |
|
|
Placebo Comparator placebo |
Saline will be administered 13 times |
|
Recruiting Locations
Orange 5379513, California 5332921 92868
Palo Alto 5380748, California 5332921 94304
Denver 5419384, Colorado 5417618 80045
Gainesville 4156404, Florida 4155751 32608
Iowa City 4862034, Iowa 4862182 52242
Kansas City 4273837, Kansas 4273857 66205
Baltimore 4347778, Maryland 4361885 21205
Worcester 4956184, Massachusetts 6254926 01655
Rochester 5134086, New York 5128638 14642
Durham 4464368, North Carolina 4482348 27708
Columbus 4509177, Ohio 5165418 43221
Philadelphia 4560349, Pennsylvania 6254927 19104
San Antonio 4726206, Texas 4736286 78229
Richmond 4781708, Virginia 6254928 23298
More Details
- NCT ID
- NCT07038200
- Status
- Recruiting
- Sponsor
- Avidity Biosciences, Inc.
Detailed 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.