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NCT05747924 Clinical Trial

Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)

Muscular Dystrophies Clinical Trial

— FORTITUDE  

Official title:
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 Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT05747924
Other study ID # AOC 1020-CS1
Secondary ID 2022-002704-2020
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date April 4, 2023
Est. completion date September 2025

Study information
Verified date February 2024
Source Avidity Biosciences, Inc.
Contact Amy Halseth, PhD
Phone 858-771-7038
Email medinfo@aviditybio.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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 Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Description:

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, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in adult participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2). Part A is a dose titration design which includes a single and multiple dose schedule with 1 cohort. Part B is a single-ascending and multiple-ascending dose design with 2 cohorts. Part C is a parallel, multi-dose cohort design with 1 cohort. For each of Part A, B, and C the patient duration is 12 months as the active treatment period is approximately 9 months followed by a 3-month follow-up period. Once participants have completed active treatment and the follow-up period, they may have the option to participate in a planned open-label extension. If patients do not enroll in the open-label extension, they will be followed through a 6-month safety follow-up period.

Recruitment information / eligibility
Status Recruiting
Enrollment 72
Est. completion date September 2025
Est. primary completion date September 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years to 65 Years
Eligibility Inclusion Criteria: - FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor) - Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces) - At least 1 muscle region suitable for biopsy (testing provided by Sponsor) - Muscle weakness in both upper and lower body, as determined by Investigator Exclusion Criteria: - Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeeding - Unwilling or unable to continue to comply with contraceptive requirements - Body mass index (BMI) >35.0 kg/m2 at Screening - History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study - History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy - Anticipated survival less than 2 years - Blood or plasma donation within 16 weeks of Study Day 1 - Any contraindication to MRI - Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study - Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
• Placebo
Placebo will be administered via intravenous (IV) infusion

Locations
Country Name City State
Canada University of Ottawa Ottawa Ontario
United Kingdom University College London London
United Kingdom University of Sheffield Sheffield
United States Rare Disease Research Atlanta Georgia
United States Ohio State University Columbus Ohio
United States University of Texas Southwestern Dallas Texas
United States University of Colorado Denver Colorado
United States Duke University Durham North Carolina
United States University of Florida Gainesville Florida
United States Kansas University Medical Center Kansas City Kansas
United States University of California Los Angeles Los Angeles California
United States Stanford University Palo Alto California
United States University of Pennsylvania Philadelphia Pennsylvania
United States Virginia Commonwealth University Richmond Virginia
United States University of Rochester Medical Center Rochester New York
United States University of California San Diego San Diego California
United States University of Washington Seattle Washington

Sponsors (1)
Lead Sponsor Collaborator
Avidity Biosciences, Inc.

Countries where clinical trial is conducted

United States,  Canada,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Incidence of treatment-emergent adverse events Through study completion, up to Day 365
Secondary Plasma pharmacokinetic (PK) parameters of AOC 1020 Observed maximum concentration Through study completion; up to Day 365
Secondary Plasma pharmacokinetic (PK) parameters of AOC 1020 Observed half-life Through study completion; up to Day 365
Secondary Plasma pharmacokinetic (PK) parameters of AOC 1020 Observed area under the curve Through study completion; up to Day 365
Secondary Muscle drug concentration Concentration of siRNA component in skeletal muscle Day 120
Secondary Urine drug concentration Fraction of drug excreted in urine 0 - 24 hours after first and third dose
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