Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy (FSHD)
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ClinicalTrials.gov Identifier: NCT02603562 |
Recruitment Status :
Completed
First Posted : November 13, 2015
Last Update Posted : May 17, 2017
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Condition or disease | Intervention/treatment | Phase |
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Facioscapulohumeral Muscular Dystrophy (FSHD) | Biological: ATYR1940 Biological: Placebo | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 8 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 Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy |
Actual Study Start Date : | March 30, 2016 |
Actual Primary Completion Date : | December 13, 2016 |
Actual Study Completion Date : | February 14, 2017 |

Arm | Intervention/treatment |
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Experimental: ATYR1940
Intrapatient dose escalation ATYR1940: ATYR1940 will be administered as an IV infusion at doses of 0.3, 1.0, and 3.0 mg/kg for up to 12 Weeks. The dose level in this study will not exceed 3.0 mg/kg.
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Biological: ATYR1940
ATYR1940 will be administered as an IV infusion at doses of 0.3, 1.0, and 3.0 mg/kg using intrapatient dose escalation. The dose level in this study will not exceed 3.0 mg/kg |
Placebo Comparator: Placebo
An initial IV infusion of placebo will be supplied as normal saline, and administered over a 30-minute period at Week 1.
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Biological: Placebo
An initial IV infusion of placebo will be supplied as normal saline, and administered over a 30-minute period at Week 1. |
- Incidences of Treatment-Emergent adverse events and serious adverse events [ Time Frame: 12 weeks ]Incidences of adverse events including serious and severe adverse events overall and by intensity
- Changes from Baseline in safety laboratory test results [ Time Frame: Changes from Baseline after 12 weeks ]Changes from Baseline in safety laboratory test results
- Changes from Baseline in pulmonary evaluation [ Time Frame: Changes from Baseline after 12 week ]Change from Baseline in pulmonary evaluation
- Changes from Baseline in visual assessment [ Time Frame: Changes from Baseline after 12 weeks ]Changes in Baseline in visual acuity
- Changes from Baseline in hearing [ Time Frame: Changes from Baseline after 12 weeks ]Safety Primary Outcome Measure - Changes from Baseline in hearing based on audiometry
- Immunogenicity Outcome Measure - Incidence and level of ADA [ Time Frame: 12 weeks ]Incidence and level of ADA titers
- Immunogenicity Outcome Measure - Incidence and level of Jo-1 Ab [ Time Frame: 12 weeks ]Incidence and level of Jo-1 Ab titers
- Immunogenicity Outcome Measure - Incidence of infusion reactions [ Time Frame: 12 weeks ]Incidence of infusion reactions
- Pharmacodynamic Additional Outcome Measure - Changes in FSHD-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 from baseline clinical parameters [ Time Frame: 12 weeks ]Changes from baseline in muscle strength based on manual muscle strength

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Ages Eligible for Study: | 12 Years to 25 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Established, genetically confirmed diagnosis of FSHD.
- Onset of FSHD signs or symptoms prior to 10 years of age, as documented in the patient's medical record or based on patient or family report.
- Provide written informed consent or assent
- In the Investigator's opinion, patient is willing and able to complete all study procedures and comply with the weekly study visit schedule.
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 or activity 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 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): NCT02603562
United States, California | |
Stanford University | |
Stanford, California, United States, 94305 | |
United States, Iowa | |
University of Iowa Children's Hospital | |
Iowa City, Iowa, United States, 52242 | |
United States, Ohio | |
OSU Wexner Medical Center | |
Columbus, Ohio, United States, 43210 | |
United States, Utah | |
University of Utah | |
Salt Lake City, Utah, United States, 84132 | |
France | |
Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET) | |
Marseille, France, 13385 | |
Institut de Myologie | |
Paris, France, 75651 | |
Italy | |
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | |
Milano, Italy, 20133 |
Study Director: | Kelly Blackburn | aTyr Pharma |
Responsible Party: | aTyr Pharma, Inc. |
ClinicalTrials.gov Identifier: | NCT02603562 |
Other Study ID Numbers: |
ATYR1940-C-003 2014-003346-27 ( EudraCT Number ) |
First Posted: | November 13, 2015 Key Record Dates |
Last Update Posted: | May 17, 2017 |
Last Verified: | May 2017 |
FSHD |
Muscular Dystrophies Muscular Dystrophy, Facioscapulohumeral Muscular Disorders, Atrophic Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |