Phase 1/2 Study of AOC 1020 in Adults with Facioscapulohumeral Muscular Dystrophy (FSHD)
Research type
Research Study
Full title
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 Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
IRAS ID
1007580
Contact name
Teresa Brandt
Contact email
Sponsor organisation
Avidity Biosciences Inc.
Clinicaltrials.gov Identifier
Research summary
Trial design \nRandomized: Study participants will be randomly assigned (like flipping a coin) to receive either active drug (AOC 1020) or placebo. A placebo looks like the active drug but does not contain AOC 1020. \nDouble blind: Neither study participants nor study doctor will know if study participants are receiving the active drug or a placebo. \nThis study has 3 parts (Part A, B, C). Each part will require patient participation for approximately 13.5 months, followed by either (1) a 6-month safety follow-up period or (2) an open label extension study of AOC 1020 (All study participants will receive active drug).\nTrial population \nAdults aged 18-65 years of age with FSHD type 1 or FSHD type 2 who have mild to moderate symptoms (able to walk 10 meters with or without assistive devices, muscle weakness in both upper and lower body as determined by the investigator), at least 1 muscle region suitable for biopsy and testing that identifies the FSHD-causing gene mutation(s) are being asked to participate in this study. \nPeople that are pregnant or breastfeeding, have a body mass index (BMI) greater than 35, bleeding disorders, skin or muscle conditions that would be unsuitable for serial muscle biopsy, for whom magnetic resonance imaging (MRI) would not be a safe procedure, whose recent laboratory test results are not normal or other conditions that would be considered medically unsuitable for this study will be excluded. \nInterventions and Duration \nStudy participants will receive a total of 5 doses of study drug (either active AOC 1020 or placebo) over a 9-month period. Drug will be given by intravenous infusion (through a needle directly into the blood stream). Participants will 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, muscle biopsies, physical function tests and health questionnaires.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
23/SC/0104
Date of REC Opinion
10 May 2023
REC opinion
Further Information Favourable Opinion