FSHD: Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy
Study Details
Study Description
Brief 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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 1/Phase 2 |
Detailed Description
Study ATYR1940-C-006 is a multi-national, multi-center, open-label extension study designed to evaluate the long-term safety, effects on muscle, and PD of ATYR1940 in patients with LGMD2B or FSHD previously treated in the Protocol ATYR1940-C-003 (Stage 1 only) or ATYR1940-C-004 (i.e., the parent studies). This study will be conducted at the same study centers at which patients were enrolled in the parent studies.
Patients who completed the treatment period in the parent study; in the Investigator's opinion, demonstrated acceptable tolerability of ATYR1940, are considered by the Investigator to be compliant with ATYR1940 and the study procedures, and do not meet any criterion for ATYR1940 discontinuation are eligible for participation in the current study, contingent upon Investigator and patient agreement to continue ATYR1940 treatment.
For the first 12 weeks in this extension study, patients will receive ATYR1940 at the highest tolerated dose received in the parent study; no dose adjustments are allowed during this 12-week period. After 12 weeks, if the patient is demonstrating good tolerability, the ATYR1940 dose may be increased on a patient-specific basis at the Investigator's discretion, in consultation with the Sponsor and Medical Monitor. ATYR1940 dose increases to >3.0 mg/kg are not permissible.
All patients will receive ATYR1940 on a weekly basis in this study, regardless of the frequency of dosing in the parent study. ATYR1940 will be administered via IV infusion over 90 minutes. If medically indicated, the infusion duration and volume may be adjusted at the Investigator's discretion in consultation with the Medical Monitor and Sponsor.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ATYR1940 All patients will receive ATYR1940 at the highest tolerated dose received in the parent study for 12-weeks. After 12 weeks, if the patient is demonstrating good tolerability, theATYR1940 dose may be increased on a patient-specific basis at the Investigator's discretion, in consultation with the Sponsor and Medical Monitor. ATYR1940 dose increases to >3.0 mg/kg are not permissible. |
Drug: ATYR1940
ATYR1940 at a dose no greater than 3.0 mg/kg administered via IV infusion over 90 minutes once weekly.
|
Outcome Measures
Primary Outcome Measures
- Incidences of Treatment-Emergent adverse events and serious adverse events [12-weeks]
Incidences of adverse events including serious and severe adverse events overall and by intensity
- Changes from Baseline in safety laboratory test results [12-weeks]
Changes from Baseline in safety laboratory test results
- Changes from Baseline in pulmonary evaluation of PFTs [12-weeks]
Change from Baseline in pulmonary evaluation of PFTs
- Change in Baseline in ECG findings [12-weeks]
Change in Baseline in 12-lead electrocardiogram findings
- Change from Baseline in vital signs [12-weeks]
Change from Baseline in vital signs
- Immunogenicity Outcome Measure - Incidence of ADA [12-weeks]
Incidence of ADA titers
- Immunogenicity Outcome Measure - Incidence of Jo-1 Ab [12-weeks]
Incidence of Jo-1 Ab titers
- Change from Baseline in Muscle strength using MMT [12-weeks]
Change from Baseline in muscle strength using MMT
- Changes from Baseline in pulmonary evaluation of pulse oximetry [12-weeks]
Change from Baseline in pulmonary evaluation of pulse oximetry
Secondary Outcome Measures
- Change from Baseline in CK levels [12-weeks]
Change from Baseline in CK levels
Eligibility Criteria
Criteria
Inclusion Criteria:
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Enrolled in and completed the treatment period in the parent study.
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Demonstrated, in the Sponsor's and Investigator's opinions, acceptable tolerability of ATYR1940.
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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.
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Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued ATYR1940 treatment.
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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:
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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).
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Planned to receive any vaccination during study participation.
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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.
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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.
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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.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California, Irvine, ALS and Neuromuscular Center | Irvine | California | United States | 92697 |
| 2 | Stanford University | Stanford | California | United States | 94305 |
| 3 | University of Utah | Salt Lake City | Utah | United States | 84132 |
| 4 | Rigshospitalet, University of Copenhagen | Copenhagen | Denmark | ||
| 5 | Foundation IRCCS Neurological Institute Carlo | Milan | Italy | 20133 |
Sponsors and Collaborators
- aTyr Pharma, Inc.
Investigators
- Study Director: Gennyne Walker, aTyr Pharma, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ATYR1940-C-006