AOC 1020 for Facioscapulohumeral Muscular Dystrophy

(FORTITUDE-OLE Trial)

Enrolling by invitation at 16 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Avidity Biosciences, Inc.

Disqualifiers: Pregnancy, Breastfeeding, New conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 22 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called AOC 1020 for individuals with Facioscapulohumeral Muscular Dystrophy (FSHD), a condition that weakens muscles in the face, shoulders, and upper arms. The main goal is to determine if AOC 1020 is safe and effective over a long period. Participants will receive the treatment intravenously, directly into the vein, 16 times over 22 months. Suitable candidates have already completed a previous study with AOC 1020 without major issues. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

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

Research shows that AOC 1020 is generally well-tolerated by patients. In earlier studies, researchers tested AOC 1020 in adults with facioscapulohumeral muscular dystrophy (FSHD) to assess its safety and tolerability. These studies primarily focused on identifying any serious side effects. The results indicated that most participants did not experience major problems with the treatment.

Some individuals did encounter mild to moderate side effects, but these were manageable. No major safety concerns emerged that would prevent further study of the treatment. Thus, AOC 1020 is considered safe enough for ongoing clinical research. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Facioscapulohumeral Muscular Dystrophy, which mainly focus on managing symptoms like inflammation and muscle pain, AOC 1020 works using an innovative approach. It targets the genetic causes of the disease through a new mechanism of action, potentially slowing down or even stopping muscle degeneration. Researchers are excited about its unique delivery method, as it is administered intravenously, allowing for precise dosing over an extended period. This approach could offer a more effective and long-lasting treatment option compared to current therapies.

What evidence suggests that AOC 1020 might be an effective treatment for Facioscapulohumeral Muscular Dystrophy?

Research has shown that AOC 1020, also known as Del-brax, offers promising results for treating facioscapulohumeral muscular dystrophy (FSHD). In earlier studies, patients who received AOC 1020 demonstrated better muscle function than those given a placebo. These improvements were significant compared to the typical progression of the disease. This suggests that AOC 1020 may enhance muscle strength and function in individuals with FSHD. ^{1 2 3 4 5}

Who Is on the Research Team?

Amy Halseth, Ph.D.

Principal Investigator

Avidity Biosciences, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with Facioscapulohumeral Muscular Dystrophy (FSHD), a type of muscular dystrophy that affects the muscles of the face, shoulder blades, and upper arms. Participants should have completed prior studies with AOC 1020 or meet specific health criteria.

Inclusion Criteria

Completion of AOC 1020-CS1 with no significant tolerability issues and satisfactory compliance with protocol requirements

Ability to provide written informed consent and comply with all study requirements

Exclusion Criteria

Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding

I don't have new or worsening conditions that would stop me from completing the study.

I cannot or will not follow the birth control rules during the AOC 1020-CS2 study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Treatment

Participants receive AOC 1020 intravenously every 6 to 7 weeks for a total of 16 doses over 22 months

22 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, including monitoring for adverse events, concomitant medications, and pregnancy status

12 weeks

Open-label extension

Continuation of treatment to evaluate long-term safety, tolerability, and efficacy of AOC 1020

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• AOC 1020

Trial Overview The study tests long-term safety and effectiveness of AOC 1020 when given through an IV. It's an open-label extension, meaning everyone knows they're getting AOC 1020 and there's no placebo group.

How Is the Trial Designed?

Group I: AOC 1020 Regimen Experimental Treatment 1 Intervention

AOC 1020 Dose Regimen; Sixteen doses administered intravenously over 22 months. All participants will receive AOC 1020 at a dose level of 2mg/kg.

AOC 1020 is already approved in United States for the following indications:

Approved in United States as AOC 1020 for:

• Facioscapulohumeral muscular dystrophy (FSHD)

Who Is Running the Clinical Trial?

Avidity Biosciences, Inc.

Lead Sponsor

Trials

8

Recruited

960+

Published Research Related to This Trial

The study involved 55 affected individuals and 48 at-risk individuals from a large family with facioscapulohumeral muscular dystrophy (FSHMD), showing that their clinical symptoms are similar to those seen in chromosome 4-linked FSHMD.

Genetic analyses revealed no linkage to known genetic markers for FSHMD on chromosome 4, suggesting that this family has a different genetic cause for their condition, supporting the idea of genetic heterogeneity in FSHMD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Studies in Non-chromosome 4-Linked Facioscapulohumeral Muscular Dystrophy. Tim, RW., Gilbert, JR., Stajich, JM., et al.[2019]

In a study of 96 patients with facioscapulohumeral muscular dystrophy (FSHD), six early-onset cases were identified, indicating that early-onset FSHD can occur both in familial and sporadic forms.

The early-onset cases exhibited significant variability in symptoms, including progression of muscle weakness and associations with hearing loss and retinopathy, suggesting that early-onset FSHD is part of a broader clinical spectrum rather than a distinct form of the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Facioscapulohumeral muscular dystrophy in early childhood. Brouwer, OF., Padberg, GW., Wijmenga, C., et al.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07038200

A Study to Evaluate Del-brax (Also Referred to as AOC ... ... Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD). Detailed Description. The study consists of a ...

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

Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf FORTITUDE (AOC 1020-CS1) is a randomized, placebo-controlled, double-blind, global trial designed to evaluate the safety and tolerability of del-brax in adult ...

3. 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 ... Notably, treated patients demonstrated improved muscle function compared with both placebo and data from the ReSolve natural history study.

4. trialx.com trialx.com/clinical-trials/listings/299701/a-study-to-evaluate-del-brax-also-referred-to-as-aoc-1020-in-participants-with-fshd/

A Study to Evaluate Del-brax (Also Referred to as ... Overview. A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the ...

5. practicalneurology.com practicalneurology.com/news/investigational-therapy-for-facioscapulohumeral-muscular-dystrophy-shows-promise-according-to-avidity-biosciences/2470511/

Investigational Therapy for Facioscapulohumeral Muscular ... ... AOC 1020 as a treatment for 39 adult participants with FSHD. The primary outcomes are safety and tolerability, with additional endpoints ...