84 Participants Needed

AOC 1020 for Facioscapulohumeral Muscular Dystrophy

(FORTITUDE-OLE Trial)

Recruiting at 16 trial locations
Age: Any Age
Sex: Any
Trial Phase: Phase 2
Sponsor: Avidity Biosciences, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

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.

What makes the drug AOC 1020 unique for treating facioscapulohumeral muscular dystrophy?

AOC 1020 (delpacibart braxlosiran, del-brax) is unique because it represents a novel approach to treating facioscapulohumeral muscular dystrophy, a condition with limited standard treatment options. Its specific mechanism of action, ingredients, or administration route may differ from existing therapies, but detailed information is not provided in the available research.12345

What is the purpose of this trial?

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)

Research Team

AH

Amy Halseth, Ph.D.

Principal Investigator

Avidity Biosciences, Inc.

Eligibility Criteria

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

Ability to provide written informed consent and comply with all study requirements
Completion of AOC 1020-CS1 with no significant tolerability issues and satisfactory compliance with protocol 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

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

Treatment Details

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.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: AOC 1020 RegimenExperimental Treatment1 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)

Find a Clinic Near You

Who Is Running the Clinical Trial?

Avidity Biosciences, Inc.

Lead Sponsor

Trials
8
Recruited
960+

Findings from Research

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

References

Clinical Studies in Non-chromosome 4-Linked Facioscapulohumeral Muscular Dystrophy. [2019]
Facioscapulohumeral muscular dystrophy with EcoRI/BlnI fragment size of more than 32 kb. [2019]
Facioscapulohumeral muscular dystrophy in early childhood. [2019]
[Clinical and molecular diagnosis of facioscapulohumeral dystrophy type 1 (FSHD1) in 2012]. [2013]
FSH dystrophy 4q35 deletion in patients presenting with facial-sparing scapular myopathy. [2019]
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