Clinical trial NCT02531217: Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients With Fascioscapulohumeral Muscular Dystrophy (FSHD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Facioscapulohumeral Muscular Dystrophy
Lead sponsor: aTyr Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2015-08-24
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies.

Detailed description: The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies, and to additionally explore the pharmacokinetics and biologic activity of ATYR1940 in adult participants with molecularly defined genetic muscular dystrophies. Participants who successfully complete the parent study (NCT02239224) are eligible for enrollment into this long-term extension study.

Eligibility criteria: Inclusion Criteria:

* Participant has an established, genetically-confirmed, diagnosis of facioscapulohumeral dystrophy with clinical findings meeting existing criteria.
* Participant is a male or female aged 18 to 65 years, inclusive.
* Participants who previously participated in study ATYR1940-C-002 and who meet the entry criteria above for the current study will be eligible for enrollment.

Exclusion Criteria:

* Participant is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (for example, etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or non-steroidal anti-inflammatory agents (NSAIDs) within 2 weeks before Baseline.
* Participant has a severe retinopathy.
* Participant 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.
* Participant has 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.
* Participant has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.
* If female and of childbearing potential (premenopausal and not surgically sterile), participant has a positive pregnancy test at Screening or is unwilling to use contraception from the time of Screening through the 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
* If male, participant is unwilling to use a condom plus spermicide during sexual intercourse from the time of Screening through the 1-month Follow-up visit.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: Up to End of Study (up to Week 36)
measure: Number of Participants With Clinically Significant Physical Examination Abnormality
timeFrame: Up to End of Study (up to Week 36)
measure: Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
timeFrame: Up to End of Study (up to Week 36)
measure: Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE
timeFrame: Up to End of Study (up to Week 36)
measure: Number of Participants With a Clinical Laboratory Abnormality Resulting in an AE
timeFrame: Up to End of Study (Up to Week 36)
Secondary outcomes:
Other outcomes:
Locations:
city: Columbus
country: United States
facility: aTyr Pharma Investigative Site
state: Ohio
city: Rome
country: Italy
facility: aTyr Pharma Investigative Site
city: Nijmegen
country: Netherlands
facility: aTyr Pharma Investigative Site
Study officials:
References: