Overview

This trial has been completed.

Conditions limb-girdle muscular dystrophies, facioscapulohumeral muscular dystrophy
Treatments atyr1940, placebo
Phase phase 1/phase 2
Sponsor aTyr Pharma, Inc.
Start date September 2015
End date October 2016
Trial size 18 participants
Trial identifier NCT02579239, 2015-001910-88, ATYR1940-C-004

Summary

The purpose of this study is to assess the safety and biological activity of ATYR1940 in patients with Limb Girdle (LGMD2B) or 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 of intravenous ATYR1940 administered twice weekly at doses of 0.3, 1.0, or 3.0 mg/kg for up to 12 weeks.
atyr1940
Intrapatient dose escalation of intravenous ATYR1940 administered twice weekly at doses of 0.3, 1.0, or 3.0 mg/kg for up to 12 weeks.
(Placebo Comparator)
Patients will receive an initial infusion of placebo at Week 1, supplied as normal saline and administered via IV infusion over a 30-minute period.
placebo
Patients will receive an initial infusion of placebo at Week 1, supplied as normal saline and administered via IV infusion over a 30-minute period.

Primary Outcomes

Measure
Tolerability Primary Outcome Measure - incidence of treatment-emergent adverse events (AEs) and serious adverse events overal (SAEs) overall and by intensity
time frame: 12 weeks
Safety Primary Outcome Measure - change from Baseline in safety laboratory test results
time frame: 12 weeks
Safety Primary Outcome Measure - change from Baseline in ECG
time frame: 12 weeks
Safety Primary Outcome Measure - change from Baseline in vital signs measurements and pulmonary evaluations
time frame: 12 week

Secondary Outcomes

Measure
Pharmacodynamic Additional Outcome Measure - changes in muscular dystrophy-related inflammatory immune state
time frame: 12 weeks
Pharmacodynamic Additional Outcome Measure - changes in serum- and/or plasma-based muscle biomarkers.
time frame: 12 weeks

Eligibility Criteria

Male or female participants from 18 years up to 75 years old.

Inclusion Criteria: - Provided informed consent - Investigator's opinion, patient is willing and able to complete all study procedures and comply with the study visit schedule. Patients with LGMD2B: - Established, genetically confirmed diagnosis of LGMD2B. - Either the presence of a STIR-positive muscle on lower extremity skeletal muscle MRI, or, if no STIR-positive muscles, meets muscle biomarker criteria. Patients with FSHD: - Established, genetically confirmed diagnosis of FSHD. - The presence of a STIR-positive muscle on lower extremity skeletal muscle MRI. 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 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 LGMD2B or 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 Limb Girdle and Facioscapulohumeral Muscular Dystrophies
Description ATYR1940-C-004 is a Phase1b/2 open-label intrapatient study dose escalation study aiming to evaluate the safety, tolerability, immunogenicity, biological activity of intravenous ATYR1940, administered once weekly for 8 weeks, then twice a weekly for 4 weeks in adult patients with LGMD2B and FSHD. Approximately 8 LGMD2B and 8 FHSD patients will be enrolled.
Trial information was received from ClinicalTrials.gov and was last updated in December 2016.
Information provided to ClinicalTrials.gov by aTyr Pharma, Inc..