Facioscapulohumeral muscular dystrophy MedDRA version: 19.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
aTyr Pharma, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Enrolled in =Cohort 2 and completed the double-blind treatment period in the parent study. 2. Demonstrated, in the Investigator’s opinion, acceptable tolerability of study drug. 3. In the Investigator’s opinion, patient has shown acceptable compliance with study drug and the study procedures in the parent study and is willing and able to comply with all procedures in the current study. 4. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued study drug treatment. 5. Provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures. Are the trial subjects under 18? no 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 32 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. At any time during participation in the parent study, met a study drug discontinuation criterion, including, but not limited to: a. Jo-1 Ab levels =1.5 U/mL. b. Clinical evidence of a generalized infusion-related reaction (IRR). c. Clinical evidence of a National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) =Grade 2, study-drug-related SAE. d. Pregnancy. e. Progression of disease that, in the opinion of the Investigator, precluded further participation in the study. f. Withdrawal of consent. g. Other findings that, at the discretion of the Investigator and/or Sponsor, indicated that study drug administration should be discontinued. 2. Is expected to require treatment with curcumin or systemic albuterol (intermittent inhaled albuterol is permissible) during study participation; or 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, creatine, L-carnitine) on a chronic basis; or statin treatment initiation or significant adjustment to statin regimen (stable, chronic statin use is permissible). 3. Planned to receive any vaccination during study participation. 4. Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator’s opinion, might jeopardize the patient's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study. 5. Evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation. 6. If female and of childbearing potential (premenopausal and not surgically sterile), has a positive pregnancy test at entry or is unwilling to use contraception from the time of entry through the 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device. 7. If male, is unwilling to use a condom plus spermicide during sexual intercourse from the time of entry through the 1 month Follow-up visit"
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical study ATYR1940-C-002;Secondary Objective: • Evaluate the effects of long-term ATYR1940 treatment on clinically relevant measures of muscle strength and function, including: Quantitative Muscle Testing (QMT) and Manual muscle testing (MMT), as determined by the Investigator • Evaluate the effects of long-term ATYR1940 treatment on patient-reported quality of life (QoL) • Evaluate the effects of long-term ATYR1940 treatment on muscle disease burden, based on skeletal muscle magnetic resonance imaging (MRI) • Explore biological and pharmacodynamic (PD) changes in the inflammatory immune state in peripheral blood • Determine the long-term systemic exposure to ATYR1940 through pharmacokinetic (PK) sampling;Primary end point(s): Safety and tolerability: • Change from baseline in physical examination, including neurological examination. • Incidence of AEs, including serious and severe AEs. • Change from baseline in safety laboratory test results. • Change from baseline in electrocardiogram (ECG) findings. • Change from baseline in vital sign measurements and pulmonary evaluations (pulmonary function tests and pulse oximetry). • Anti-drug antibody (ADA) titers and Jo-1 antibody levels. • Exploratory characterization of immune response to ATYR1940.;Timepoint(s) of evaluation of this end point: Physical Exam: at eligibility check, W4, every 3M and at 1 and 4-week FU AEs: weekly and at 1, 4 and 12-week FU Safety lab tests: at eligibility check, W1 to W4, monthly and at 1, 4 and 12-week FU ECG: at W1, W2, W4, every 3M and at 1 and 4-week FU Vital signs: at eligibility check, weekly and at 1, 4 and 12-week FU Pulmonary function tests: at W1, every 3M and at 4-week FU Pulse Oximetry: at W1 to W4, every 3M and at 1, 4 and 12-week FU Jo-1 Ab: at eligibility ch | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Muscle strength and function: • Muscle strength, based on Quantitative muscle testing (QMT) and Manual muscle testing (MMT) • Lower extremity muscle function based on the Vignos scale Quality of life: • INQoL questionnaire • FSHD-HI questionnaire • Additional measures of quality of life (e.g., sleep status) Pharmacodynamic effects of ATYR1940: • Muscle disease burden on lower extremity skeletal muscle MRI • Changes in FSHD-related inflammatory immune state in peripheral blood Systemic exposure: • PK sampling;Timepoint(s) of evaluation of this end point: QMT & MMT & Vignos scale & INQoL & sleep status: at eligibility check, every 3M and at 1-week FU FSHD-HI: at eligibility check and every 3M Skeletal Muscle Surveillance MRI: at eligibility check, at M3 and then every 6M and at 4-week FU PBMCs & biomarkers: at eligibility check, at W4, every 3M and at 1 and 4-week FU Pharmacokinetics: at W1 to W4, every 3M and at 1-week FU | — |
Countries
Italy, Netherlands, United States
Contacts
Public ContactClinical Trial Operations
Voisin Consulting
Outcome results
None listed