FSHDPhase 1/2FORTITUDENCT05747924
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 1/2 study testing AOC 1020 in people with FSHD and FSHD1, run by Avidity Biosciences, Inc. at 17 sites in 3 countries.
Trial summary
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) Read more about this study
- Who is it for?
- Ages 16 to 70 years
- What is being tested?
- AOC 1020
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 17 sitesin 3 countries
- Study dates
- 4 Apr 2023to 20 Mar 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
- Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
- At least 1 muscle region suitable for biopsy (testing provided by Sponsor)
- Muscle weakness in both upper and lower body, as determined by Investigator
Who cannot join
- Pregnant or intends to become pregnant while on study, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
- Body mass index (BMI) >35.0 kg/m2 at Screening
- History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
- History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
- Anticipated survival less than 2 years
Show all 10Show fewer
- Blood or plasma donation within 16 weeks of Study Day 1
- Any contraindication to MRI
- Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study
- Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening
Trial locations and contacts
17 sites in 3 countries.
17 sites in 3 countries
United States14 sites
Rare Disease Research
Atlanta, Georgia, 30329
Ohio State University
Columbus, Ohio, 43221
University of Texas Southwestern
Dallas, Texas, 75390
University of Colorado
Denver, Colorado, 80045
Duke University
Durham, North Carolina, 27708
University of Florida
Gainesville, Florida, 32608
Kansas University Medical Center
Kansas City, Kansas, 66205
University of California Los Angeles
Los Angeles, California, 90095
Stanford University
Palo Alto, California, 94304
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Rochester Medical Center
Rochester, New York, 14642
University of California San Diego
San Diego, California, 92093
University of Washington
Seattle, Washington, 98104
United Kingdom2 sites
University College London
London, WIT 7HA
University of Sheffield
Sheffield, S10 2TN
About this study
Official 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 Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
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)
Read the detailed description
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.
- Being tested
- AOC 1020, Placebo
- Keywords
- FORTITUDE, Avidity, Avidity Biosciences, AOC 1020
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral, Facioscapulohumeral Muscular Dystrophy 1B, Muscular Dystrophies, Facioscapulohumeral muscular dystrophy 1a
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AOC 1020Drug
AOC 1020 will be administered via intravenous (IV) infusion
PlaceboDrug
Also called Saline
Placebo will be administered via intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| AOC 1020 Regimen 1 | Experimental | Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months Receives: Drug: AOC 1020 |
| AOC 1020 Regimen 2 | Experimental | Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months Receives: Drug: AOC 1020 |
| AOC 1020 Regimen 3 | Experimental | Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months Receives: Drug: AOC 1020 |
| Placebo (Saline) Regimen 1 | Placebo comparator | Cohort A \& B: Placebo; Five doses administered intravenously over 9 months Receives: Drug: Placebo |
| Placebo (Saline) Regimen 2 | Placebo comparator | Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (Cohorts A & B)
Measured: Through study completion, up to Day 365
Change in plasma KHDC1L (Part C)
Ratio to Baseline
Measured: Across months 3 to 12
Secondary outcomes (5)
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Observed maximum concentration
Measured: Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Observed half-life
Measured: Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Observed area under the curve
Measured: Through study completion; up to Day 365
Muscle drug concentration (Cohorts A & B)
Concentration of siRNA component in skeletal muscle
Measured: Day 120
Change in circulating creatine kinase (Cohort C)
Ratio to Baseline
Measured: Across months 3 to 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2023
- Study start4 Apr 2023
- Primary completion18 Mar 2026
- Study completion20 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FSHDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05747924
- Sponsor's protocol ID
- AOC 1020-CS1
- Acronym
- FORTITUDE
- Status last verified
- Jul 2026
- First submitted
- 17 Feb 2023
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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 Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
- ClinicalTrials.gov ID
- NCT05747924
- Acronym / sponsor ID
- FORTITUDE / AOC 1020-CS1
- Keywords
- FORTITUDE, Avidity, Avidity Biosciences, AOC 1020
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Apr 2023 (actual)
- Primary completion
- 18 Mar 2026 (actual)
- Study completion
- 20 Mar 2026 (actual)
- First posted
- 28 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| AOC 1020 Regimen 1 | Experimental | Drug: AOC 1020Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months |
| AOC 1020 Regimen 2 | Experimental | Drug: AOC 1020Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months |
| AOC 1020 Regimen 3 | Experimental | Drug: AOC 1020Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months |
| Placebo (Saline) Regimen 1 | Placebo comparator | Drug: PlaceboCohort A \& B: Placebo; Five doses administered intravenously over 9 months |
| Placebo (Saline) Regimen 2 | Placebo comparator | Drug: PlaceboCohort C: Placebo; Eight doses administered intravenously over approximately 10 months |
Who may qualify
- Ages
- 16 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
- Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
- At least 1 muscle region suitable for biopsy (testing provided by Sponsor)
- Muscle weakness in both upper and lower body, as determined by Investigator
Key exclusion criteria (10 listed)
- Pregnant or intends to become pregnant while on study, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
- Body mass index (BMI) >35.0 kg/m2 at Screening
- History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
- History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AOC 1020AOC 1020 will be administered via intravenous (IV) infusion | Drug | AOC 1020 Regimen 1, AOC 1020 Regimen 2, AOC 1020 Regimen 3 |
| PlaceboAlso: SalinePlacebo will be administered via intravenous (IV) infusion | Drug | Placebo (Saline) Regimen 1, Placebo (Saline) Regimen 2 |
Primary outcome
Incidence of treatment-emergent adverse events (Cohorts A & B)
Time frame: Through study completion, up to Day 365
Change in plasma KHDC1L (Part C)
Time frame: Across months 3 to 12
Ratio to Baseline
5 secondary outcomes
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Time frame: Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Time frame: Through study completion; up to Day 365
Plasma pharmacokinetic (PK) parameters of AOC 1020 (Cohorts A & B)
Time frame: Through study completion; up to Day 365
Muscle drug concentration (Cohorts A & B)
Time frame: Day 120
Change in circulating creatine kinase (Cohort C)
Time frame: Across months 3 to 12
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2026.
- Avidity Biosciences WebsiteLink provided by the sponsor.
- FORTITUDE Study WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for FSHD
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD
Facioscapulohumeral Muscular Dystrophy (FSHD)
RecruitingObservational study
Biomarker Development for Muscular Dystrophies
Myotonic Dystrophy, Duchenne Muscular Dystrophy
RecruitingPhase 1
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD - Facioscapulohumeral Muscular Dystrophy
Not yet recruitingPhase 2
A Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral Muscular Dystrophy (FSHD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AOC 1020 in people with FSHD and FSHD1, run by Avidity Biosciences, Inc. at 17 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, United Kingdom, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (Cohorts A & B) (time frame: Through study completion, up to Day 365). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2026.