Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy

• Status: Completed
• Trial end date: 2017-04-18
• Target enrollment: 0
• Participant gender: All

Summary

ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in the Study ATYR1940-C-003 (Stage 1 only) or ATYR1940-C-004 (i.e., the parent studies).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: aTyr Pharma, Inc.

Criteria

Inclusion Criteria:

1. Enrolled in and completed the treatment period in the parent study.

2. Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940.

3. In the Investigator's opinion, patient has shown acceptable compliance with ATYR1940 and the study procedures in the parent study and is willing and able to comply with all procedures in the current study.

4. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued ATYR1940 treatment.

5. Provide written informed consent or assent after the nature of the study has been explained and prior to the performance of any research-related procedures.

Exclusion Criteria:

1. Is expected to require treatment with curcumin or systemic albuterol (intermittent inhaled albuterol is permissible) during study participation; or use of a product that putatively enhances muscle growth (e.g., insulin-like growth factor, growth hormone) or activity (e.g., Coenzyme Q, Coenzyme A, creatine, L-carnitine) on a chronic basis; or statin treatment initiation or significant adjustment to statin regimen (stable, chronic statin use is permissible).

2. Planned to receive any vaccination during study participation.

3. Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator's opinion, might jeopardize the patient's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.

4. 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.

5. If female and of childbearing potential (premenopausal and not surgically sterile), has a positive pregnancy test at entry or is unwilling to use contraception from the time of entry through the 3-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.

6. If male, is unwilling to use a condom plus spermicide during sexual intercourse from the time of entry through the 1 month Follow-up visit.