A Phase 2 Extension Study to Evaluate AOC 1020 in Adult Participants with FSHD
Research type
Research Study
Full title
A Phase 2 Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, and Pharmacokinetics of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
IRAS ID
1010368
Contact name
Nirvana Moodley
Contact email
Sponsor organisation
Avidity Biosciences Inc.
Clinicaltrials.gov Identifier
Research summary
This is an extension study for participants with Facioscapulohumeral Muscular Dystrophy (FSHD) who were previously enrolled in the AOC 1020-CS1 clinical study. The treatment offered in this study is designed to reduce the amount of DUX4 protein (the protein that causes FSHD) in muscle. The primary objective is to evaluate the long-term safety and tolerability of the study drug, called AOC 1020, in participants with FSHD. Participants from the Phase 1/2 AOC 1020-CS1 study will continue to receive study drug via quarterly IV infusions. All participants who enroll in this study will receive the active drug, AOC 1020, regardless of whether they received the active drug or the placebo in AOC 1020-CS1.
The total duration of active treatment in this study is approximately 24 months. Once participants have completed active treatment, they will be followed for a 9-month safety follow-up period.
The study will include adult participants with FSHD who have completed the AOC 1020-CS1 clinical study and have consented to also receive treatment in this study.
People that intend to or become pregnant or are breastfeeding, or who have a new or worsening medical conditions that would be considered medically unsuitable for this study will be excluded.
Study participants will receive a total of 9 doses of the active drug, AOC 1020, over a 2-year period. Drug will be given by intravenous infusion (through a needle directly into the blood stream). Participants will continue to be monitored for safety and tolerability, and for the ability of the study drug to impact various measures of FSHD. Assessments and procedures will involve physical exams, blood, and urine tests, MRI, physical function tests and health questionnaires.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
24/EE/0231
Date of REC Opinion
5 Sep 2024
REC opinion
Further Information Favourable Opinion