FSHDPhase 2FORTITUDE-OLENCT06547216
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 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 Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and 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 72 years
- What is being tested?
- AOC 1020
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 84 participants
- Where
- 17 sitesin 3 countries
- Study dates
- 25 Jul 2024to Jun 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 72 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
- Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:
- No significant tolerability issues
- Satisfactory compliance with the AOC 1020-CS1 protocol requirements
Who cannot join
- Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
- Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
- Any new conditions or worsening of existing condition(s) 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. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)
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 Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Read the detailed description
This study will continue to evaluate the safety, tolerability, and efficacy of AOC 1020 in participants who were treated in the randomized, placebo-controlled, Phase 1/2 AOC 1020-CS1 clinical study.
Participants from AOC 1020-CS1 are eligible to enroll in AOC 1020-CS2 if they have satisfactorily completed AOC 1020-CS1. All participants who enroll in AOC 1020-CS2 will receive AOC 1020 regardless of whether they received AOC 1020 or placebo in AOC 1020-CS1. Dosing will occur every 6 to 7 weeks (twice quarterly).
The total duration of active treatment in AOC 1020-CS2 is approximately 46 months. Once participants have completed active treatment, they will be followed through a 18-week safety follow-up period to monitor for adverse events (AEs), concomitant medications, and pregnancy status (as applicable). Total duration on study is up to approximately 50 months (4 years 2 month), excluding the potential 8-week screening period.
- Being tested
- AOC 1020
- Keywords
- FORTITUDE, Avidity, Avidity Biosciences, AOC 1020, FORTITUDE-OLE, FORTITUDE OLE
- 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
Also called delpacibart braxlosiran, del-brax
AOC 1020 will be administered via intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| AOC 1020 Regimen | Experimental | AOC 1020 Dose Regimen; Thirty-two doses administered intravenously over 46 months. All participants will receive AOC 1020 at a dose level of 2mg/kg. Receives: Drug: AOC 1020 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events
Measured: Through study completion, up to Day 729
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2024
- Study start25 Jul 2024
- Primary completionApr 2030 (estimated)
- Study completionJun 2030 (estimated)
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 84 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06547216
- Sponsor's protocol ID
- AOC 1020-CS2
- Acronym
- FORTITUDE-OLE
- Status last verified
- Jul 2026
- First submitted
- 7 Aug 2024
- Last updated
- 14 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 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
- ClinicalTrials.gov ID
- NCT06547216
- Acronym / sponsor ID
- FORTITUDE-OLE / AOC 1020-CS2
- Keywords
- FORTITUDE, Avidity, Avidity Biosciences, AOC 1020, FORTITUDE-OLE, FORTITUDE OLE
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 84 participants (estimated)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 Jul 2024 (actual)
- Primary completion
- Apr 2030 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 9 Aug 2024 (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 Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Detailed description
This study will continue to evaluate the safety, tolerability, and efficacy of AOC 1020 in participants who were treated in the randomized, placebo-controlled, Phase 1/2 AOC 1020-CS1 clinical study.
Participants from AOC 1020-CS1 are eligible to enroll in AOC 1020-CS2 if they have satisfactorily completed AOC 1020-CS1. All participants who enroll in AOC 1020-CS2 will receive AOC 1020 regardless of whether they received AOC 1020 or placebo in AOC 1020-CS1. Dosing will occur every 6 to 7 weeks (twice quarterly).
The total duration of active treatment in AOC 1020-CS2 is approximately 46 months. Once participants have completed active treatment, they will be followed through a 18-week safety follow-up period to monitor for adverse events (AEs), concomitant medications, and pregnancy status (as applicable). Total duration on study is up to approximately 50 months (4 years 2 month), excluding the potential 8-week screening period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AOC 1020 Regimen | Experimental | Drug: AOC 1020AOC 1020 Dose Regimen; Thirty-two doses administered intravenously over 46 months. All participants will receive AOC 1020 at a dose level of 2mg/kg. |
Who may qualify
- Ages
- 16 to 72 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
- Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:
Key exclusion criteria (3 listed)
- Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
- Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
- Any new conditions or worsening of existing condition(s) 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. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)
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 1020Also: delpacibart braxlosiran, del-braxAOC 1020 will be administered via intravenous (IV) infusion | Drug | AOC 1020 Regimen |
Primary outcome
Incidence of treatment-emergent adverse events
Time frame: Through study completion, up to Day 729
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2026.
- Avidity Biosciences WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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 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 3 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: under way, not looking for participants, as of the sponsor's last update on 14 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
This study is still under way. Results are not available in the source record yet.
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 (time frame: Through study completion, up to Day 729). 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 14 Jul 2026.