A Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies

Phase 1

• Conditions: imb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy; MedDRA version: 19.0Level: PTClassification code 10064087Term: Facioscapulohumeral muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2015-001910-88-DK
• Lead Sponsor: aTyr Pharma, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-06-30
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

1. Male or female aged 18 to 75 years, inclusive.
2. Provided written informed consent.
3. In the Investigator’s opinion, is willing and able to complete all study procedures and comply with the study visit schedule.
4. Established genetically confirmed diagnosis of LGMD2B or FSHD.
5. Either the presence of a STIR positive muscle with limited fatty infiltration, as determined by the Fischer scoring system, on MRI (FHSD) or an elevated muscle marker (LGMD2B)
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 12
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion Criteria

1. Currently receiving treatment with an immunomodulatory agent or history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) or corticosteroids within the 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
2. Currently receiving curcumin or albuterol or requires such treatment during study participation; use of a product that putatively enhances muscle growth or activity on a chronic basis within 30 days before baseline; or statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline.
3. Use of an investigational product or device (other than a mobility assistance device) within 30 days before baseline.
4. History of severe restrictive or obstructive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence of active lung disease on screening chest radiograph.
5. History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or Jo 1 Ab level =0.6 U/mL on screening.
6. Acute or clinically relevant Epstein-Barr virus (EBV) or cytomegalovirus (CMV) infection or re-activation.
7. Chronic infection, such as hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) or a history of tuberculosis.
8. Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
9. Symptomatic cardiomyopathy or severe cardiac arrhythmia (including bradyarrythmias), that may, in the Investigator’s opinion, limit the patient’s ability to complete the study protocol.
10. Gamma-glutamyl transferase (GGT) or serum creatinine levels >2× the upper limit of normal.
11. Muscle biopsy within 30 days before baseline.

Study & Design

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