Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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16 December 2017 |
Main ID: |
NCT02603562 |
Date of registration:
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05/11/2015 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy
FSHD |
Scientific title:
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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 |
Date of first enrolment:
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March 30, 2016 |
Target sample size:
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8 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT02603562 |
Study type:
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Interventional |
Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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France
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Italy
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United States
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Contacts
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Name:
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Kelly Blackburn |
Address:
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Telephone:
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Email:
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Affiliation:
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aTyr Pharma |
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Key inclusion & exclusion criteria
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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.
Age minimum:
12 Years
Age maximum:
25 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Facioscapulohumeral Muscular Dystrophy (FSHD)
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Intervention(s)
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Biological: Placebo
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Biological: ATYR1940
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Primary Outcome(s)
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Changes from Baseline in pulmonary evaluation
[Time Frame: Changes from Baseline after 12 week]
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Changes from Baseline in hearing
[Time Frame: Changes from Baseline after 12 weeks]
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Changes from Baseline in safety laboratory test results
[Time Frame: Changes from Baseline after 12 weeks]
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Changes from Baseline in visual assessment
[Time Frame: Changes from Baseline after 12 weeks]
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Incidences of Treatment-Emergent adverse events and serious adverse events
[Time Frame: 12 weeks]
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Secondary Outcome(s)
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Immunogenicity Outcome Measure - Incidence and level of ADA
[Time Frame: 12 weeks]
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Immunogenicity Outcome Measure - Incidence and level of Jo-1 Ab
[Time Frame: 12 weeks]
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Immunogenicity Outcome Measure - Incidence of infusion reactions
[Time Frame: 12 weeks]
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Secondary ID(s)
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ATYR1940-C-003
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2014-003346-27
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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