A Study to Evaluate the Safety, Tolerability, Immunogenicity and Biological Activity of ATYR1940 in Patients With Pediatric Onset FHSD
- Conditions
- Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular DystrophyMedDRA version: 20.0Level: PTClassification code 10064087Term: Facioscapulohumeral muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2014-003346-27-IT
- Lead Sponsor
- ATYR PHARMA, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 8
1. Male or female, between the ages of 16 and 25 years, inclusive, in Stage 1 and 12 and 15 years, inclusive, in Stage 2.
2. Established, genetically confirmed diagnosis of FSHD (Type 1 or 2).
3. Onset of FSHD signs or symptoms (e.g., facial weakness, scapular winging, weakness in upper or lower extremities) prior to 10 years of age, as documented in the patient's medical record or based on patient or family report.
4. Provide written informed consent or assent (as appropriate, based on age of majority).
5. In the Investigator's opinion, patient is willing and able to complete all study procedures and comply with the weekly study visit schedule
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Currently receiving treatment with an immunomodulatory agent or history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) within the 3 months before baseline; corticosteroids within 3 months before baseline; or high-dose nonsteroidal
anti-inflammatory agents (NSAIDs; either chronic or intermittent) within 2 weeks before baseline.
2. Currently receiving curcumin or albuterol or requires such treatment during study participation; 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, creatinine, L-carnitine) on a chronic basis within 4 weeks before baseline; statin treatment initiatio or significant adjustment to statin regimen within 3 months before baseline (stable, chronic statin use is permissible).
3. Use of an investigational product or device (other than a mobility assistance device) within 30 days before baseline.
4. Evidence of an alternative diagnosis other than FSHD or a co-existing myopathy or dystrophy, based on prior muscle biopsy or other available investigations.
5. History of severe restrictive or obstructive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on screening chest radiograph.
6. History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or Jo 1 Ab level = 0.6 U/mL on screening.
7. Acute or clinically relevant Epstein-Barr virus (EBV) or cytomegalovirus (CMV) infection or re-activation.
8. Chronic infection, such as hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) or a history of tuberculosis.
9. Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
10. Evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, cognitive impairment, or a condition that requires immediate surgical intervention or other treatment, or may not allow safe participation in the study.
11. Muscle biopsy within 30 days before baseline.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method