Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension

Phase 1

Completed

• Conditions: Facioscapulohumeral Muscular Dystrophy
• Interventions: Biological: ATYR1940

• Registration Number: NCT02531217
• Lead Sponsor: aTyr Pharma, Inc.

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.

Study Timeline

• Study First Submitted: 2015-06-25
• Study Start: 2015-08-13
• Study First Submitted QC: 2015-08-21
• Study First Posted: 2015-08-24
• Primary Completion: 2016-05-26
• Study Completion: 2016-05-26
• Status Verified: 2023-07
• Results First Submitted: 2023-07-25
• Results First Submitted QC: 2023-07-25
• Last Update Submitted: 2023-07-25
• Results First Posted: 2023-08-18
• Last Update Posted: 2023-08-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ATYR1940	ATYR1940	Participants will receive ATYR1940 3.0 milligrams per kilograms (mg/kg) intravenous (IV) infusion once weekly for 24 weeks.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Up to End of Study (up to Week 36)	TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs ware defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section.
Number of Participants With Clinically Significant Physical Examination Abnormality	Up to End of Study (up to Week 36)	Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat, cardiovascular system, respiratory system, gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, reflexes, and sensory testing. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Number of Participants With a Clinical Laboratory Abnormality Resulting in an AE	Up to End of Study (Up to Week 36)	Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count with differential \[neutrophils, lymphocytes, monocytes, eosinophils, basophils\], and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol \[nonfasting\]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Number of Participants With a Vital Sign-Related Event Resulting in a TEAE	Up to End of Study (up to Week 36)	The vital sign parameters that were evaluated included blood pressure, pulse and respiration rates, and body temperature. Vital signs abnormalities that were considered by the Investigator to be clinically significant were reported as AEs if the finding represented a change from baseline. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE	Up to End of Study (up to Week 36)	Pulmonary evaluations included pulmonary function tests. Clinically significant changes were to be reported as adverse events. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Trial Locations

Locations (1)

aTyr Pharma Investigative Site; 🇳🇱 Nijmegen, Netherlands