AOC 1020 for Facioscapulohumeral Muscular Dystrophy
(FORTITUDE Trial)
Recruiting in Palo Alto (17 mi)
+20 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Avidity Biosciences, Inc.
Must not be taking: Investigative medications
Disqualifiers: Pregnancy, BMI >35, Bleeding disorders, others
Trial Summary
What is the purpose of this trial?A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
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 have taken any investigative medication within 1 month before the screening.
What makes the drug AOC 1020 unique for treating facioscapulohumeral muscular dystrophy?
Currently, there is no established consensus on medication treatments for facioscapulohumeral muscular dystrophy, making AOC 1020 a potentially novel option. Other treatments like albuterol and vitamins have shown some benefits, but more clinical trials are needed to determine the effectiveness of new drugs like AOC 1020.
12345Eligibility Criteria
Adults with confirmed FSHD1 or FSHD2 who can walk at least 10 meters and have at least one muscle region suitable for biopsy. They should not be pregnant, breastfeeding, or planning pregnancy soon, must follow contraceptive guidelines, and have a BMI under 35.0 kg/m^2. Those with recent biopsies, bleeding disorders, severe muscle wasting, or conditions that could affect study participation are excluded.Inclusion Criteria
I have muscle weakness in both my arms and legs.
My FSHD (type 1 or 2) is confirmed by genetic testing.
I can walk 10 meters on my own or with a cane, stick, or braces.
+1 more
Exclusion Criteria
Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding
Blood or plasma donation within 16 weeks of Study Day 1
Unwilling or unable to continue to comply with contraceptive requirements
+7 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive AOC 1020 or placebo intravenously. Cohorts A & B receive five doses over 9 months, and Cohort C receives eight doses over approximately 10 months.
9-10 months
Follow-up
Participants are monitored for safety and effectiveness after treatment. Cohorts A & B have a 12-week follow-up, and Cohort C has a 7-week follow-up.
7-12 weeks
Open-label extension (optional)
Participants may opt into continuation of treatment long-term after completing the main trial.
Participant Groups
The trial is testing AOC 1020 against a placebo in adults with Facioscapulohumeral Muscular Dystrophy (FSHD). It's randomized and double-blind meaning neither the participants nor the researchers know who gets the real treatment versus placebo. The study will assess safety, tolerability of doses given intravenously and any potential benefits on muscle function.
5Treatment groups
Experimental Treatment
Placebo Group
Group I: AOC 1020 Regimen 3Experimental Treatment1 Intervention
Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months
Group II: AOC 1020 Regimen 2Experimental Treatment1 Intervention
Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
Group III: AOC 1020 Regimen 1Experimental Treatment1 Intervention
Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
Group IV: Placebo (Saline) Regimen 1Placebo Group1 Intervention
Cohort A \& B: Placebo; Five doses administered intravenously over 9 months
Group V: Placebo (Saline) Regimen 2Placebo Group1 Intervention
Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months
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
Research Locations NearbySelect from list below to view details:
University of MinnesotaMinneapolis, MN
University of UtahSalt Lake City, UT
University of OttawaOttawa, Canada
Duke UniversityDurham, NC
More Trial Locations
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Who Is Running the Clinical Trial?
Avidity Biosciences, Inc.Lead Sponsor
References
Facioscapulohumeral muscular dystrophy functional composite outcome measure. [2020]We developed an evaluator-administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD-COM) comprising patient-identified areas of functional burden for future clinical trials.
Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD): A Systematic Review. [2023]Facioscapulohumeral muscular dystrophy (FSHD) is the third most common type of muscular dystrophy. This disease presents as a slowly progressive asymmetric muscle weakness that involves the facial, scapular, and upper arm muscles mainly. Currently, there is no established consensus on this disease treatment in terms of medications. We assessed the response to the treatment of the drugs utilized in clinical trials by performing a systematic literature review in English using the preferred reporting items for systematic reviews (PRISMA) and meta-analyses. We only used human clinical trials in patients diagnosed with FSHD that received consistent pharmacological treatment. We included 11 clinical trials that fulfilled our criteria. We concluded that albuterol had statistically significant results in three out of four clinical trials, with improved elbow flexors muscle strength. Vitamin C, vitamin E, zinc gluconate, and selenomethionine showed significant improvement in the maximal voluntary contraction and endurance limit time of quadriceps muscle. At the same time, diltiazem and MYO-029 demonstrate no improvement in function, strength, or muscle mass. Losmapimod, currently in phase I of the ReDUX4 trial, showed promising results. Peradventure, more clinical trials are still needed to address this subject. Nevertheless, this review provides a clear and concise update on the treatment for this disease.
Facioscapulohumeral muscular dystrophy in early childhood. [2019]To determine the occurrence and the clinical and genetic variability of early-onset facioscapulohumeral muscular dystrophy (FSHD).
Clinical Studies in Non-chromosome 4-Linked Facioscapulohumeral Muscular Dystrophy. [2019]To characterize clinically and molecularly a large, non-chromosome 4-linked facioscapulohumeral muscular dystrophy (FSHMD) family.
Early onset facioscapulohumeral dystrophy - a systematic review using individual patient data. [2018]Infantile or early onset is estimated to occur in around 10% of all facioscapulohumeral dystrophy (FSHD) patients. Although small series of early onset FSHD patients have been reported, comprehensive data on the clinical phenotype is missing. We performed a systematic literature search on the clinical features of early onset FSHD comprising a total of 43 articles with individual data on 227 patients. Additional data from four cohorts was provided by the authors. Mean age at reporting was 18.8 years, and 40% of patients were wheelchair-dependent at that age. Half of the patients had systemic features, including hearing loss (40%), retinal abnormalities (37%) and developmental delay (8%). We found an inverse correlation between repeat size and disease severity, similar to adult-onset FSHD. De novo FSHD1 mutations were more prevalent than in adult-onset FSHD. Compared to adult FSHD, our findings indicate that early onset FSHD is overall characterized by a more severe muscle phenotype and a higher prevalence of systemic features. However, similar as in adults, a significant clinical heterogeneity was observed. Based on this, we consider early onset FSHD to be on the severe end of the FSHD disease spectrum. We found natural history studies and treatment studies to be very scarce in early onset FSHD, therefore longitudinal studies are needed to improve prognostication, clinical management and trial-readiness.