Overview

This trial is active, not recruiting.

Condition facioscapulohumeral muscular dystrophy (fshd)
Treatments atyr1940, placebo
Phase phase 1/phase 2
Sponsor aTyr Pharma, Inc.
Start date November 2015
End date April 2017
Trial size 8 participants
Trial identifier NCT02603562, 2014-003346-27, ATYR1940-C-003

Summary

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
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.
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)
An initial IV infusion of placebo will be supplied as normal saline, and administered over a 30-minute period at Week 1.
placebo
An initial IV infusion of placebo will be supplied as normal saline, and administered over a 30-minute period at Week 1.

Primary Outcomes

Measure
Incidences of Treatment-Emergent adverse events and serious adverse events
time frame: 12 weeks
Changes from Baseline in safety laboratory test results
time frame: Changes from Baseline after 12 weeks
Changes from Baseline in pulmonary evaluation
time frame: Changes from Baseline after 12 week
Changes from Baseline in visual assessment
time frame: Changes from Baseline after 12 weeks
Changes from Baseline in hearing
time frame: Changes from Baseline after 12 weeks

Secondary Outcomes

Measure
Immunogenicity Outcome Measure - Incidence and level of ADA
time frame: 12 weeks
Immunogenicity Outcome Measure - Incidence and level of Jo-1 Ab
time frame: 12 weeks
Immunogenicity Outcome Measure - Incidence of infusion reactions
time frame: 12 weeks

Eligibility Criteria

Male or female participants from 12 years up to 25 years old.

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.

Additional Information

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
Description 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.
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by aTyr Pharma, Inc..