A Phase 3 Study to Evaluate AOC 1020 in Participants with FSHD

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Avidity Biosciences Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

13 Apr 2026 → ongoing

Decision date (initial)

2025-11-04

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Avidity Biosciences inc

External identifiers

EU CT number

2025-521012-18-00

WHO UTN

U1111-1318-4362

ClinicalTrials.gov

NCT07038200

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Pharmacokinetic, Pharmacodynamic

To evaluate the efficacy of AOC 1020 on functional mobility

Secondary objectives 3

1. 1. To evaluate the efficacy of AOC 1020 on functional mobility and muscle strength in the upper and lower extremities
2. 2. To evaluate the efficacy of AOC 1020 on muscle strength, functional mobility, and patient-reported outcome measures
3. 3. To evaluate the effects of AOC 1020 on FSHD circulating biomarkers

Conditions and MedDRA coding

Facioscapulohumeral Muscular Dystrophy (FSHD)

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, European Medicines Agency, European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
2. 2. Male and females with FSHD confirmed by documented genetic diagnosis as defined below: - FSHD1 - FSHD2
3. 3. 18 to 70 years of age at time of informed consent
4. 4. Able to walk independently at pre-specified walking speed (orthoses or ankle braces are allowed) for at least 10 meters at screening.

Exclusion criteria 14

1. 1. Females who are pregnant, breastfeeding, or planning to become pregnant during the study period or
2. 2. Males or females not willing to comply with the contraceptive requirements
3. 3. Screening laboratory results or any other clinically significant abnormalities in screening laboratory values that would render a participant unsuitable for inclusion.
4. 4. BP > 140/90 mmHg at Screening
5. 5. Anticipated survival less than 2 years
6. 6. Evidence of current or chronic infection with hepatitis C, hepatitis B, or HIV (including chronic infection requiring ongoing treatment to maintain viral suppression)
7. 7. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1 or ongoing symptoms related to recent infection causing impairment to ADL in the opinion of the Investigator
8. 8. Malignancy within 5 years, except for basal or squamous cell carcinoma, melanoma in situ of the skin, carcinoma in situ of the cervix, or other malignancies within 5 years that have been treated with curative intent and which are not expected to recur
9. 9. Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer; or participation or plan to participate in another interventional study with an investigational drug or device or other types of interventional studies (including those studying behavioral modification and/or physical therapy) while on study
10. 10. Treatment with an oligonucleotide within 9 months of Screening
11. 11. Blood or plasma donation within 16 weeks of Study Day 1
12. 12. Recent history of or current drug or alcohol abuse in the opinion of the Investigator
13. 13. History of multiple drug allergies or history of allergic reaction to any component of, or excipient in, the Study Drug
14. 14. Presence or history of clinically significant illness, medical condition, or abnormal test result/finding that, in the opinion of the Investigator, could affect a participant’s safety or their ability to comply with study procedures and/or complete the study visit schedule. This may include, but is not limited to, a history of cardiovascular or central nervous system disease, neuromuscular diseases other than FSHD (e.g., myopathy, neuropathy, neuromuscular junction disorders), a cardiopulmonary condition, or a clinically significant mental disorder

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from Baseline to Week 78 in 10MWRT velocity (m/sec)

Secondary endpoints 2

1. 1.1 Key Secondary Endpoints • Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) velocity o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN)
2. 1.2 Other Secondary Endpoints • Change from Baseline over time in: o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity (PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Sleep Disturbance o DUX4-regulated plasma KHDC1L o Serum CK

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Avidity Biosciences Inc.

Sponsor organisation

Avidity Biosciences Inc.

Address

3020 Callan Road

City

San Diego

Postcode

92121-1109

Country

United States

Scientific contact point

Organisation

Avidity Biosciences Inc.

Contact name

Avidity Biosciences Inc

Public contact point

Organisation

Avidity Biosciences Inc.

Contact name

Avidity Biosciences Inc

Third parties 9

Locations

6 EU/EEA countries · 20 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 49 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications