Trial Search Results

Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early 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).

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

aTyr Pharma, Inc.

Stanford Investigator(s):

Intervention(s):

  • Biological: ATYR1940
  • Biological: Placebo

Phase:

Phase 1/Phase 2

Eligibility


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.

Ages Eligible for Study

12 Years - 25 Years

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Jennie Perez
650-497-3080
Recruiting