A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic Myopathy

Phase 1

• Conditions: Facioscapulohumeral muscular dystrophy; MedDRA version: 17.1Level: PTClassification code 10064087Term: Facioscapulohumeral muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2014-001753-17-NL
• Lead Sponsor: aTyr Pharma, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-06-03
• Study Completion: 2017-01-04
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Patient is a male or female aged 18 to 65 years, inclusive.
2. Patient has an established, genetically-confirmed, diagnosis of FSHD with clinical findings meeting existing criteria.
3. Patient has provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
4. Cohorts =2 only: Patient has imaging findings meeting defined criteria for muscle inflammation in at least 1 skeletal muscle (as per MRI Procedural Manual).
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 44
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patient is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or high-dose non-steroidal anti-inflammatory agents (NSAIDs) (either chronic or intermittent) within 2 weeks before Baseline.
2. Patient has evidence of an alternative diagnosis other than FSHD, based on prior muscle biopsy or genetic test findings.
3. Patient has a presumptive diagnosis of FSHD, based on clinical assessment, but does not yet have genetic confirmation of the diagnosis.
4. Patient has a history of obstructive or restrictive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on Screening chest radiograph.
5. Patient has a history of anti-synthetase syndrome, prior Jo-1 antibody (Ab)-positivity, or has a positive or equivocally positive Jo-1 Ab test result during Screening.
6. Patient has symptomatic cardiomyopathy or severe cardiac arrhythmia that may, in the Investigator’s opinion, limit the patient’s ability to complete the study protocol.
7. Patient has evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation.
8. Patient has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.
9. Patient underwent muscle biopsy within 30 days before Baseline.
10. If female and of childbearing potential (premenopausal and not surgically sterile), patient has a positive pregnancy test at Screening or is unwilling to use contraception.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified