A Phase 1/2 interventional study of AOC 1020 and Placebo in FSHD, FSHD1 and FSHD2, sponsored by Avidity Biosciences, Inc.. Completed at 17 sites in 3 countries. Open to participants aged 16 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-07-16.
Sponsored by Avidity Biosciences, Inc. · Phase 1/2, Interventional, and Treatment
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)
AOC 1020-CS1 is a first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2).
Cohort A comprises a placebo-controlled dose titration cohort (Cohort A1) which includes a nested single and multiple dose schedule. Cohort B comprises a placebo-controlled, nested single ascending dose (SAD)/multiple ascending dose (MAD) cohort (Cohort B1). Cohort C comprises a randomized, placebo-controlled, expansion cohort (Cohort C1). For each of Cohorts A, B, and C the study duration is 12 months as the active treatment period is approximately 9 months for Cohorts A \& B and approximately 10.5 months for Cohort C followed by a 12-week follow-up period for Cohorts A \& B and a 7-week follow-up period for Cohort C. Once participants have completed active treatment with follow-up through 12 months, they may have the option to participate in a planned open-label extension. If patients do not immediately roll over into the open-label extension study or decline participation, they will be followed for 18 weeks after their last dose of study medication.
74 studies on the registry are indexed under Muscular Dystrophy, Facioscapulohumeral; 25 are open to participants now.
This study's enrollment of 90 is above the median of 30 across 45 interventional studies indexed under Muscular Dystrophy, Facioscapulohumeral.
Browse Muscular Dystrophy, Facioscapulohumeral studies →Avidity Biosciences, Inc. is the lead sponsor of 11 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
Drug: AOC 1020
Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
Drug: AOC 1020
Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months
Drug: AOC 1020
Cohort A \& B: Placebo; Five doses administered intravenously over 9 months
Drug: Placebo
Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months
Drug: Placebo
AOC 1020 will be administered via intravenous (IV) infusion
Placebo will be administered via intravenous (IV) infusion
Also known as: Saline
Incidence of treatment-emergent adverse events (Cohorts A & B)
Time frame: Through study completion, up to Day 365
Change in plasma KHDC1L (Part C)
Ratio to Baseline
Time frame: Across months 3 to 12
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Observed maximum concentration
Time frame: Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Observed half-life
Time frame: Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Observed area under the curve
Time frame: Through study completion; up to Day 365
Muscle drug concentration (Cohorts A & B)
Concentration of siRNA component in skeletal muscle
Time frame: Day 120
Change in circulating creatine kinase (Cohort C)
Ratio to Baseline
Time frame: Across months 3 to 12
Plan to share: No
This study is completed, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Muscular Dystrophy, Facioscapulohumeral→
Avidity Biosciences, Inc.