AOC 1020 for FSHD

(FORTITUDE-3 Trial)

Recruiting at 21 trial locations

Overseen By Avidity Biosciences, Inc.

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Avidity Biosciences, Inc.

Must not be taking: Investigational drugs, Oligonucleotides

Disqualifiers: Pregnancy, Hypertension, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 13

Duration: 72 weeks

200 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AOC 1020 for individuals with Facioscapulohumeral Muscular Dystrophy (FSHD). The trial aims to evaluate the treatment's effectiveness and safety. Participants will receive either the actual treatment or a placebo (a harmless solution with no active treatment) through an IV. Suitable candidates have a confirmed FSHD diagnosis and can walk ten meters independently, possibly using support like braces. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you've taken another investigational drug within a month or an oligonucleotide within 9 months before the screening.

Is there any evidence suggesting that AOC-1020 is likely to be safe for humans?

Research has shown that AOC 1020 demonstrated promising safety results in earlier studies. Participants who received AOC 1020 did not encounter major safety issues, and the treatment was generally well-tolerated. Some mild side effects occurred, but they were not serious.

Since this trial is in Phase 3, earlier studies have already assessed safety. Phase 3 trials typically include more participants to confirm earlier findings. The FDA has granted Fast Track status to the treatment, often given when early data indicates potential benefits and manageable risks.

Overall, evidence suggests that AOC 1020 is safe for humans, but this trial will provide more detailed information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for FSHD?

Unlike the standard treatments for facioscapulohumeral muscular dystrophy (FSHD), which primarily focus on managing symptoms and slowing progression, AOC-1020 offers a novel approach by targeting the underlying genetic cause of the disease. Researchers are excited about AOC-1020 because it uses advanced RNA technology to modify gene expression, potentially addressing the root of the problem rather than just treating symptoms. This groundbreaking mechanism could lead to more effective and long-lasting improvements for individuals with FSHD.

What evidence suggests that AOC 1020 might be an effective treatment for FSHD?

Research shows that AOC 1020, also known as del-brax, holds promise for treating facioscapulohumeral muscular dystrophy (FSHD). Studies have found that it can significantly reduce certain genes linked to FSHD by more than 50%. Participants in previous trials who received AOC 1020 demonstrated improvements in their physical abilities. The data consistently show better results in various physical tests compared to a placebo. The FDA has granted this treatment Fast Track designation, highlighting its potential effectiveness. ^{2 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with Facioscapulohumeral Muscular Dystrophy (FSHD). Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be required to have a confirmed diagnosis of FSHD.

Inclusion Criteria

I can walk by myself at a certain speed for at least 10 meters.

My muscle strength is considered adequate.

I have been diagnosed with FSHD1 or FSHD2.

Exclusion Criteria

Blood Pressure > 140/90 mmHg at Screening

Breastfeeding, pregnancy, or intent to become pregnant during the study

I do not have any health conditions that would prevent me from joining the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

6 weeks

Treatment

Participants receive an intravenous infusion of either del-brax or placebo every 6 weeks for a total of 13 doses

72 weeks

13 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Open-label extension (optional)

Eligible participants may opt into continuation of treatment long-term, pending regulatory approval

What Are the Treatments Tested in This Trial?

Interventions

• AOC-1020

Trial Overview

The study is testing the efficacy and safety of an intravenous drug called AOC-1020 compared to a placebo. It's a phase 3 trial, meaning it's in one of the final stages before potential approval for general use if proven effective and safe.

How Is the Trial Designed?

Group I: del-brax Experimental Treatment 1 Intervention

Del-brax (AOC 1020) will be administered 13 times

Group II: placebo Placebo Group 1 Intervention

Saline will be administered 13 times

Who Is Running the Clinical Trial?

Avidity Biosciences, Inc.

Lead Sponsor

Trials

8

Recruited

960+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05747924

NCT05747924 | Phase 1/2 Study of AOC 1020 in ...

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, ...

2.

aviditybiosciences.com

aviditybiosciences.com/pipeline/fshd

pipeline fshd

Del-brax data demonstrated unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable ...

3.

jettfoundation.org

jettfoundation.org/duchenne-news/avidity-biosciences-announces-positive-topline-phase-1-2-fortitude-data-demonstrating-consistent-improvement-across-multiple-functional-measures-compared-to-placebo-in-del-brax-treated-fshd-p/

Avidity Biosciences Announces Positive Topline Phase 1/2 ...

Del-brax data from the FORTITUDE study continue to demonstrate consistent reductions in a novel circulating biomarker across two cohorts at 12 months.

4.

neurologylive.com

neurologylive.com/view/fda-accelerated-approval-open-fshd-agent-del-brax-newly-initiated-phase-3-study-confirm-efficacy

FDA Accelerated Approval Open for FSHD Agent Del-Brax ...

Avidity Biosciences advances del-brax for FSHD, initiating a pivotal Phase 3 study while exploring accelerated FDA approval pathways.

5.

fshdsociety.org

fshdsociety.org/2025/06/09/avidityupdate/

Breaking News from Avidity Biosciences

The US Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

6.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000203529

Phase 1/2 Study to Evaluate the Safety, Tolerability ...

Objective: To evaluate the safety and tolerability of single and multiple ascending doses of AOC 1020 in participants with FSHD. Background:.

7.

aviditybiosciences.com

aviditybiosciences.com/sites/default/files/2025-04/Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf

Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf

"Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial." Lancet ...

8.

mdaconference.org

mdaconference.org/abstract-library/phase-1-2-trial-evaluating-aoc-1020-safety-and-pharmacokinetics-in-adults-with-facioscapulohumeral-muscular-dystrophy-fshd-fortitude-trial-design/

Phase 1/2 Trial Evaluating AOC 1020 Safety and ...

Objective: To evaluate the safety and tolerability of single and multiple ascending doses of AOC 1020 in participants with FSHD. Background: FSHD is a rare, ...