Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy



Status:Active, not recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:12 - 25
Updated:1/11/2017
Start Date:November 2015
End Date:April 2017

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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

The purpose of this study is to assess the safety and biological activity of ATYR1940 in
patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

A Phase 1b/2 open-label, intrapatient dose escalation study aiming to evaluate the safety,
tolerability, immunogenicity, biological and pharmacodynamic activity of intravenous
ATYR1940, administered once weekly for 12 weeks, in early onset FSHD patients with signs or
symptoms prior to 10 years of age. In Stage 1, up to 8 patients between the ages of 16 and
25 years will be enrolled. Stage 2 of enrollment will include patients with early onset FSHD
between the ages of 12 and 15 years.

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.
We found this trial at
5
sites
201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Russell Butterfield, MD, PhD
Phone: 801-585-7606
University of Utah Research is a major component in the life of the U benefiting...
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Columbus, Ohio 43210
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Iowa City, Iowa 52242
Principal Investigator: Katherine Mathews, MD
Phone: 319-356-2673
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Iowa City, IA
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Marseille,
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450 Serra Mall
Stanford, California 94305
(650) 723-2300
Phone: 650-497-3080
Stanford University Stanford University, located between San Francisco and San Jose in the heart of...
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Stanford, CA
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