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Facioscapulohumeral Muscular DystrophyPhase 3FORTITUDE-3NCT07038200

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

A Phase 3 study testing AOC-1020 in people with Facioscapulohumeral Muscular Dystrophy and FSHD, run by Avidity Biosciences, Inc. at 46 sites in 10 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of 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
200 participants
Where
46 sitesin 10 countries
Study dates
10 Jun 2025to Jul 2028
Phase
Phase 3
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 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Clinical and genetic diagnosis of FSHD1 or FSHD2
  • Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
  • Adequate muscle strength based on QMT composite score

Who cannot join

  • Breastfeeding, pregnancy, or intent to become pregnant during the study
  • Unwilling or unable to comply with contraceptive requirements
  • Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
  • Blood Pressure > 140/90 mmHg at Screening
  • Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
  • Treatment with an oligonucleotide within 9 months of Screening

Trial locations and contacts

46 sites in 10 countries. Contact the study team or a site near you to ask about taking part.

Avidity Biosciences, Inc., A Novartis Company contact

1-888-669-6682

novartis.email@novartis.com

Avidity Biosciences, Inc., A Novartis Company contact

+41613241111

novartis.email@novartis.com

46 sites in 10 countries

United States14 sites

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205

    Active, not recruiting

  • Ohio State University

    Columbus, Ohio, 43221

    Active, not recruiting

  • University of Colorado

    Denver, Colorado, 80045

    Active, not recruiting

  • Duke University

    Durham, North Carolina, 27708

    Active, not recruiting

  • University of Florida

    Gainesville, Florida, 32608

    Active, not recruiting

  • University of Iowa

    Iowa City, Iowa, 52242

    Active, not recruiting

  • Kansas University Medical Center

    Kansas City, Kansas, 66205

    Active, not recruiting

  • University of California Irvine

    Orange, California, 92868

    Active, not recruiting

  • Stanford University

    Palo Alto, California, 94304

    Active, not recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Active, not recruiting

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

    Active, not recruiting

  • University of Rochester Medical Center

    Rochester, New York, 14642

    Active, not recruiting

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78229

    Active, not recruiting

  • University of Massachusetts

    Worcester, Massachusetts, 01655

    Active, not recruiting

Other trials in United States

Canada4 sites, 4 recruiting

  • University of Calgary - Cumming School of Medicine

    Calgary, Alberta

    Recruiting

  • University of Alberta Hospital

    Edmonton, Alberta

    Recruiting

  • Genge Partners Inc

    Montreal, Quebec

    Recruiting

  • The Ottawa Hospital

    Ottawa, Ontario

    Recruiting

Other trials in Canada

France4 sites, 4 recruiting

  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

    Marseille

    Not yet recruiting

  • CHU de Montpellier - Hopital Gui de Chauliac

    Montpellier

    Not yet recruiting

  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires

    Nice

    Not yet recruiting

  • AP-HP Hopital Pitie-Salpetriere

    Paris

    Recruiting

Other trials in France

Germany4 sites, 4 recruiting

  • Universitaetsklinikum Bonn

    Bonn

    Recruiting

  • Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Goettingen

    Göttingen

    Recruiting

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    München

    Recruiting

  • Universitaetsklinikum Ulm

    Ulm

    Recruiting

Other trials in Germany

Italy4 sites, 4 recruiting

  • Fondazione Serena ETS - Centro Clinico NeMO Milano

    Milan

    Recruiting

  • Azienda Ospedaliero Universitaria Pisana

    Pisa

    Recruiting

  • Azienda Ospedaliero-Universitaria Sant'Andrea

    Roma

    Recruiting

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    Roma

    Recruiting

Other trials in Italy

Japan4 sites, 4 recruiting

  • National Hospital Organization Osaka Toneyama Medical Center

    Osaka

    Recruiting

  • The University of Osaka Hospital

    Osaka

    Recruiting

  • National Hospital Organization Sendai Nishitaga Hospital

    Sendai

    Recruiting

  • National Center of Neurology and Psychiatry

    Tokyo

    Recruiting

Other trials in Japan

Spain4 sites, 4 recruiting

  • Hospital Universitari de Bellvitge

    Barcelona

    Recruiting

  • Hospital Universitario Vall d'Hebron

    Barcelona

    Recruiting

  • Hospital Universitario Donostia

    San Sebastián

    Recruiting

  • Hospital Universitari i Politecnic La Fe

    Valencia

    Recruiting

Other trials in Spain

United Kingdom4 sites, 4 recruiting

  • National Hospital for Neurology & Neurosurgery

    London

    Recruiting

  • St. George's University Hospitals NHS Foundation Trust

    London

    Recruiting

  • Royal Victoria Infirmary

    Newcastle upon Tyne

    Recruiting

  • Royal Hallamshire Hospital

    Sheffield

    Recruiting

Other trials in United Kingdom

Denmark2 sites, 2 recruiting

  • Aarhus University Hospital

    Aarhus

    Not yet recruiting

  • Rigshospitalet

    Copenhagen

    Recruiting

Other trials in Denmark

Netherlands2 sites, 2 recruiting

  • Academisch Ziekenhuis Leiden

    Leiden

    Recruiting

  • Stichting Radboud Universitair Medisch Centrum

    Nijmegen

    Recruiting

Other trials in Netherlands

About this study

Official title: A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Read the detailed description

The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose).

Participants will be randomized to receive an intravenous infusion of either delbrax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.

After completion of the Week 78 visit, eligible participants will have the option to enroll in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 12 weeks for safety.

An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Being tested
AOC-1020, Placebo
Keywords
Avidity, Avidity Biosciences, del-brax, del brax, delbrax, AOC1020, AOC 1020, delpacibart braxlosiran, FORTITUDE-3, FORTITUDE Phase 3, FORTITUDE, FORTITUDE 3
Medical subject headings
Muscular Dystrophy, Facioscapulohumeral, Facioscapulohumeral Muscular Dystrophy 1B, 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 Del-brax

    Del-brax will be administered by intravenous (IV) infusion.

  • PlaceboDrug

    Also called Saline

    Placebo will be administered by intravenous (IV) infusion.

Study groups in NCT07038200
Study groupRoleWhat they receive
Del-braxExperimentalDel-brax (AOC 1020) will be administered 13 times
Receives: Drug: AOC-1020
PlaceboPlacebo comparatorSaline will be administered 13 times
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)

    Assessment of muscle strength using standardized quantitative measurements

    Measured: Baseline through Week 78

Secondary outcomes (10)

  1. Change from Baseline to Week 78 in 10MWRT

    Assessment of the time required to walk or run 10 meters as quickly and safely as possible

    Measured: Baseline through Week 78

  2. Change from Baseline to Week 78 in Timed Up-and-Go (TUG)

    Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down

    Measured: Baseline through Week 78

  3. Change from Baseline to Week 78 in Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire

    Assessment of patient-reported physical function using the PROMIS Physical Function Short Form 20a questionnaire

    Measured: Baseline through Week 78

  4. Change from Baseline to Week 78 in PROMIS Fatigue

    Assessment of patient-reported fatigue using the PROMIS Fatigue questionnaire

    Measured: Baseline through Week 78

  5. Change from Baseline to Week 78 in Worst Pain Numeric Rating Scale (NRS)

    Assessment of the severity and impact of fatigue over the previous 7 days using the self-reported PROMIS Fatigue Short Form 6a questionnaire

    Measured: Baseline through Week 78

  6. Change from Baseline to Week 78 in Patient Global Impression of Severity/Change (PGI-S/PGI-C)

    Assessment of participant-reported disease severity and change in disease status using the Patient Global Impression of Severity/Change (PGI-S/PGI-C) scales

    Measured: Baseline through Week 78

  7. Change from Baseline to Week 78 in Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function

    Assessment of health-related quality of life using the Quality of Life in Neurological Disorders (Neuro-QoL) questionnaire

    Measured: Baseline through Week 78

  8. Change from Baseline to Week 78 in NeuroQoL Sleep Disturbance

    Assessment of participant-reported sleep disturbance using the Neuro-QoL Sleep Disturbance questionnaire

    Measured: Baseline through Week 78

  9. Change from Baseline to Week 78 in DUX4-regulated plasma KHDC1L

    Assess of changes from baseline in circulating PD biomarkers relevant to FSHD

    Measured: Baseline through Week 78

  10. Change from Baseline to Week 78 in Serum CK

    Assess of changes from baseline in circulating PD biomarkers relevant to FSHD

    Measured: Baseline through Week 78

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Jun 2025
  2. Study start10 Jun 2025
  3. Primary completionMay 2028 (estimated)
  4. Study completionJul 2028 (estimated)

Resources

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
200 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07038200
Sponsor's protocol ID
AOC 1020-CS3
Acronym
FORTITUDE-3
Status last verified
Jul 2026
First submitted
13 Jun 2025
Last updated
27 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing AOC-1020 in people with Facioscapulohumeral Muscular Dystrophy and FSHD, run by Avidity Biosciences, Inc. at 46 sites in 10 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 3 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 27 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 46 sites in United States, Canada, France, Germany, Italy and 5 more countries. 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 phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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?

Contact Avidity Biosciences, Inc., A Novartis Company (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN) (time frame: Baseline through Week 78). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07038200.

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 27 Jul 2026.

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