AOC 1020 for Facioscapulohumeral Muscular Dystrophy
(FORTITUDE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you have taken any investigative medication within 1 month before the screening.
What makes the drug AOC 1020 unique for treating facioscapulohumeral muscular dystrophy?
Currently, there is no established consensus on medication treatments for facioscapulohumeral muscular dystrophy, making AOC 1020 a potentially novel option. Other treatments like albuterol and vitamins have shown some benefits, but more clinical trials are needed to determine the effectiveness of new drugs like AOC 1020.12345
Are You a Good Fit for This Trial?
Adults with confirmed FSHD1 or FSHD2 who can walk at least 10 meters and have at least one muscle region suitable for biopsy. They should not be pregnant, breastfeeding, or planning pregnancy soon, must follow contraceptive guidelines, and have a BMI under 35.0 kg/m^2. Those with recent biopsies, bleeding disorders, severe muscle wasting, or conditions that could affect study participation are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AOC 1020 or placebo intravenously. Cohorts A & B receive five doses over 9 months, and Cohort C receives eight doses over approximately 10 months.
Follow-up
Participants are monitored for safety and effectiveness after treatment. Cohorts A & B have a 12-week follow-up, and Cohort C has a 7-week follow-up.
Open-label extension (optional)
Participants may opt into continuation of treatment long-term after completing the main trial.
What Are the Treatments Tested in This Trial?
Interventions
- AOC 1020
- Placebo
AOC 1020 is already approved in United States for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Avidity Biosciences, Inc.
Lead Sponsor