Phase 2
Completed N=8
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral Muscular Dystrophy (FSHD)
Source: ClinicalTrials.gov NCT02603562 ↗
Enrolled (actual)
8
Serious AEs
0.0%
Results posted
Oct 2023
Primary outcomePrimary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 8; 0 Participants
Summary
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
8; 0 | — |
| PRIMARY Number of Participants With a Clinical Laboratory Abnormality Leading to an AE |
1 | — |
| PRIMARY Number of Participants With an Ocular Abnormality Leading to a TEAE |
— | — |
| PRIMARY Number of Participants With an Impact on Hearing From ATYR1940 Treatment |
— | — |
| PRIMARY Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE |
— | — |
| SECONDARY Number of Participants With Positive Anti-Drug Antibodies (ADA) |
4 | — |
| SECONDARY Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) |
— | — |
| SECONDARY Number of Participants With Infusion-Related Reactions |
1 | — |
| SECONDARY Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 14 |
3.8 | — |
Eligibility Criteria
Inclusion Criteria
- Established, genetically confirmed diagnosis of FSHD.
- Onset of FSHD signs or symptoms prior to 10 years of age, as documented in the participant's medical record or based on participant or family report.
- Provide written informed consent or assent
- In the Investigator's opinion, participant is willing and able to complete all study procedures and comply with the weekly study visit schedule.
Exclusion Criteria
- Currently receiving treatment with an immunomodulatory agent including targeted biological therapies within the 3 months before baseline; corticosteroids within 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
- Currently receiving curcumin or albuterol; use of a product that putatively enhances muscle growth or activity on a chronic basis within 4 weeks before baseline; statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline (stable, chronic statin use is permissible).
- Use of an investigational product or device within 30 days before baseline.
- Evidence of an alternative diagnosis other than FSHD or a coexisting myopathy or dystrophy, based on prior muscle biopsy or other available investigations.
- History of severe restrictive or obstructive lung disease, or evidence for interstitial lung disease on screening chest radiograph.
- History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or a positive or equivocally positive Jo-1 Ab test result during screening.
- Chronic infection, such as hepatitis B, hepatitis C, or human immunodeficiency virus or a history of tuberculosis.
- Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
- Symptomatic cardiomyopathy or severe cardiac arrhythmia, that may, in the Investigator's opinion, limit the participant's ability to complete the study protocol.
- Muscle biopsy within 30 days before baseline.
Data sourced from ClinicalTrials.gov (NCT02603562). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.