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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02836418
• Date of registration: 30/06/2016
• Prospective Registration: Yes
• Primary sponsor: aTyr Pharma, Inc.
• Public title: Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy FSHD
• Scientific title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy
• Date of first enrolment: July 12, 2016
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02836418
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Denmark, Italy, United States

Contacts

• Name: Gennyne Walker
• Affiliation: aTyr Pharma, Inc.

Key inclusion & exclusion 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.



Age minimum: 16 Years
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Limb Girdle Muscular Dystrophy

Facioscapulohumeral Muscular Dystrophy

Intervention(s)

Drug: ATYR1940

Primary Outcome(s)

Change from Baseline in vital signs [Time Frame: 12-weeks]

Incidences of Treatment-Emergent adverse events and serious adverse events [Time Frame: 12-weeks]

Immunogenicity Outcome Measure - Incidence of Jo-1 Ab [Time Frame: 12-weeks]

Changes from Baseline in pulmonary evaluation of pulse oximetry [Time Frame: 12-weeks]

Change from Baseline in Muscle strength using MMT [Time Frame: 12-weeks]

Change in Baseline in ECG findings [Time Frame: 12-weeks]

Changes from Baseline in pulmonary evaluation of PFTs [Time Frame: 12-weeks]

Changes from Baseline in safety laboratory test results [Time Frame: 12-weeks]

Immunogenicity Outcome Measure - Incidence of ADA [Time Frame: 12-weeks]

Secondary Outcome(s)

Change from Baseline in CK levels [Time Frame: 12-weeks]

Secondary ID(s)

ATYR1940-C-006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors