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

Completed · Phase 1/Phase 2

Conditions studied: Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy

In brief

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

Key facts

Study ID

NCT02836418

Run by

aTyr Pharma, Inc.

People needed

8

Starts

2016-07-13

Expected to finish

2017-04-18

Last updated by the study team

2023-12-22

Who can join

Age: 16 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Enrolled in and completed the treatment period in the parent study.
• Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940.
• In the Investigator's opinion, participant 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.
• Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued ATYR1940 treatment.
• 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.

You may not qualify if…

• 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 (for example, insulin-like growth factor, growth hormone) or activity (for example, 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).
• Planned to receive any vaccination during study participation.
• Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator's opinion, might jeopardize the participant's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
• Evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention, other treatment, or may not allow safe participation.
• 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.
• 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.

Where it is running

• University of California, Irvine, ALS and Neuromuscular Center — Irvine, California, United States
• Stanford University — Stanford, California, United States
• University of Utah — Salt Lake City, Utah, United States
• Rigshospitalet, University of Copenhagen — Copenhagen, Denmark
• Foundation IRCCS Neurological Institute Carlo — Milan, Italy

Full record on ClinicalTrials.gov

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