The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies (FSHD)
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ClinicalTrials.gov Identifier: NCT02579239 |
Recruitment Status :
Completed
First Posted : October 19, 2015
Last Update Posted : May 17, 2017
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Condition or disease | Intervention/treatment | Phase |
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Limb-Girdle Muscular Dystrophies Facioscapulohumeral Muscular Dystrophy | Biological: ATYR1940 Biological: Placebo | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 18 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophies |
Actual Study Start Date : | November 5, 2015 |
Actual Primary Completion Date : | October 5, 2016 |
Actual Study Completion Date : | October 5, 2016 |

Arm | Intervention/treatment |
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Experimental: ATYR1940
Intrapatient dose escalation of intravenous ATYR1940 administered twice weekly at doses of 0.3, 1.0, or 3.0 mg/kg for up to 12 weeks.
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Biological: ATYR1940
Intrapatient dose escalation of intravenous ATYR1940 administered twice weekly at doses of 0.3, 1.0, or 3.0 mg/kg for up to 12 weeks. |
Placebo Comparator: Placebo
Patients will receive an initial infusion of placebo at Week 1, supplied as normal saline and administered via IV infusion over a 30-minute period.
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Biological: Placebo
Patients will receive an initial infusion of placebo at Week 1, supplied as normal saline and administered via IV infusion over a 30-minute period. |
- Tolerability Primary Outcome Measure - incidence of treatment-emergent adverse events (AEs) and serious adverse events overal (SAEs) overall and by intensity [ Time Frame: 12 weeks ]Incidences of Adverse Events (AEs), including serious and severe AEs
- Safety Primary Outcome Measure - change from Baseline in safety laboratory test results [ Time Frame: 12 weeks ]Change from Baseline in safety laboratory test results (clinical chemistry, hematology, and urinalysis)
- Safety Primary Outcome Measure - change from Baseline in ECG [ Time Frame: 12 weeks ]Change from Baseline in 12-lead ECG findings
- Safety Primary Outcome Measure - change from Baseline in vital signs measurements and pulmonary evaluations [ Time Frame: 12 week ]Change from Baseline in vital signs measurements (blood pressure, pulse, respiratory rate, and body temperature) and pulmonary evaluations (PFTs and pulse oximetry)
- Pharmacodynamic Additional Outcome Measure - changes in muscular dystrophy-related inflammatory immune state [ Time Frame: 12 weeks ]Effects assessed by changes in muscular dystrophy-related inflammatory immune state in peripheral blood
- Pharmacodynamic Additional Outcome Measure - changes in serum- and/or plasma-based muscle biomarkers. [ Time Frame: 12 weeks ]Serum-based muscle biomarkers

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Provided informed consent
- Investigator's opinion, patient is willing and able to complete all study procedures and comply with the study visit schedule.
Patients with LGMD2B:
- Established, genetically confirmed diagnosis of LGMD2B.
- Either the presence of a STIR-positive muscle on lower extremity skeletal muscle MRI, or, if no STIR-positive muscles, meets muscle biomarker criteria.
Patients with FSHD:
- Established, genetically confirmed diagnosis of FSHD.
- The presence of a STIR-positive muscle on lower extremity skeletal muscle MRI.
Exclusion Criteria:
- Currently receiving treatment with an immunomodulatory agent, including targeted biological therapies within the 3 months before baseline; corticosteroids within 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
- Currently receiving curcumin or albuterol; use of a product that putatively enhances muscle growth on a chronic basis within 4 weeks before baseline; statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline (stable, chronic statin use is permissible).
- Use of an investigational product or device within 30 days before baseline.
- Evidence of an alternative diagnosis other than LGMD2B or FSHD or a coexisting myopathy or dystrophy, based on prior muscle biopsy or other available investigations.
- History of severe restrictive or obstructive lung disease or evidence for interstitial lung disease on screening chest radiograph.
- History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or a positive or equivocally positive Jo-1 Ab test result during screening.
- Chronic infection, such as hepatitis B, hepatitis C, or human immunodeficiency virus or a history of tuberculosis.
- Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
- Symptomatic cardiomyopathy or severe cardiac arrhythmia that may in the Investigator's opinion, limit the patient's ability to complete the study protocol.
- Muscle biopsy within 30 days before baseline.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02579239
United States, California | |
University of California, Irvine, ALS and Neuromuscular Center | |
Irvine, California, United States, 92697 | |
United States, Maryland | |
Kennedy Krieger Institute; The Johns Hopkins University School of Medicine | |
Baltimore, Maryland, United States, 21205 | |
United States, Ohio | |
OSU Wexner Medical Center | |
Columbus, Ohio, United States, 43210 | |
Denmark | |
Rigshospitalet, University of Copenhagen | |
Copenhagen, Denmark | |
France | |
Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET) | |
Marseille, France, 13385 | |
Institut de Myologie, Hôpital Pitié-Salpêtrière | |
Paris, France |
Study Director: | Kelly Blackburn | aTyr Pharma |
Responsible Party: | aTyr Pharma, Inc. |
ClinicalTrials.gov Identifier: | NCT02579239 |
Other Study ID Numbers: |
ATYR1940-C-004 2015-001910-88 ( EudraCT Number ) |
First Posted: | October 19, 2015 Key Record Dates |
Last Update Posted: | May 17, 2017 |
Last Verified: | May 2017 |
LGMD2B FSHD |
Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Muscular Dystrophy, Facioscapulohumeral Muscular Disorders, Atrophic Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |