Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: ATYR1940
Product Code: ATYR1940
Pharmaceutical Form: Concentrate for solution for infusion
Current Sponsor code: ATYR1940
Concentration unit: mg/ml milligram(s)/millilitre
Concentration
Sponsors
ATYR PHARMA, INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Enrolled in and completed the treatment period in the parent study. 2. Demonstrated, in the Investigator’s opinion, acceptable tolerability of ATYR1940. 3. In the Investigator’s opinion, patient has shown acceptable compliance with ATYR1940 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 Investigator’s and Sponsor’s opinion, a suitable candidate for continued ATYR1940 treatment. 5. Provide 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? 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 20 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 ATYR1940 discontinuation criterion, including, but not limited to: - Jo-1 antibody (Ab) levels =1.5 U/mL. - Clinical evidence of a generalized infusion-related reaction (IRR). - Clinical evidence of a National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) =Grade 2, study-drug-related serious adverse event (SAE). - Pregnancy. - Progression of disease that, in the opinion of the Investigator, precluded further participation in the study. - Withdrawal of consent. - 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. Patient 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 3-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: To evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in patients with limb girdle muscular dystrophy 2B (LGMD2B) or fascioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical study ATYR1940-C-003 (Stage 1 only) or ATYR1940-C-004.;Secondary Objective: To explore the biological and pharmacodynamic (PD) activity of ATYR1940 in patients with LGMD2B and FSHD, based on changes in: ¿ Serum-based muscle biomarkers. ¿ Inflammatory immune state in peripheral blood. ¿ Muscle disease and muscle disease burden, based on skeletal muscle MRI. ¿ Skeletal muscle strength. ¿ Upper and lower extremity muscle function. ¿ Quality of life measures.;Primary end point(s): Safety and tolerability: • Incidence of treatment-emergent adverse events (TEAEs) and SAEs overall and by intensity • Changes from baseline in: - Safety laboratory test results - Electrocardiogram (ECG) findings - Vital signs measurements - Pulmonary evaluations (pulmonary function tests [PFTs] and pulse oximetry) - Incidence and level of anti-drug antibodies (ADA) titers and Jo-1 Ab levels • Exploratory characterization of immune response to ATYR1940;Timepoint(s) of evaluation of this end point: Safety and tolerability: - Adverse events & vital signs & pulse oximetry & Jo-1 Ab: at week 1, every week and at 1, 4 and 12-week follow-up - Lab test results & ADA: at week 1, every month and at 1, 4 and 12-week follow-up - ECG: at week 1, every month and at 1 and 4-week follow-up - Pulmonary function tests: at week 1, every 6 weeks or every 3 months and at 4-week follow-up | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Muscle strength and function: ¿ Changes from baseline in the following clinical parameters: - Muscle disease burden on lower extremity skeletal muscle MRI - Muscle strength, based on manual muscle testing (MMT) - Upper and lower extremity muscle function, based on the Brooke and Vignos scales, respectively Pharmacodynamics (exploratory): ¿ Changes in muscular-dystrophy-related inflammatory immune state in peripheral blood, as assessed by: - Circulating immune proteins, such as cytokines - Ex vivo inflammatory immune protein (including cytokines) release from peripheral blood mononuclear cells (PBMCs) - Immunophenotyping of circulating PBMCs ¿ Changes in serum- and/or plasma-based muscle biomarkers Quality of life (exploratory): ¿ INQoL questionnaire ¿ For patients with FSHD, the FSHD-specific Health Inventory (FSHD-HI) questionnaire Systemic exposure: ¿ Sparse pharmacokinetic (PK) sampling;Timepoint(s) of evaluation of this end point: Muscle strength and function: - Surveillance skeletal muscle MRI: at week 1, every 3 months and at 12-week follow-up - MMT & Upper and lower extremity muscle function: at week 1, every 3 months and at 1-week follow-up Pharmacodynamics: - PBMCs & muscle biomarkers: at week 1, every 3 months and at 1 and 4-week follow-up Quality of life: - INQoL & FSHD-HI: at week 1, every 3 months and at 1 and 12-week follow-up Systemic exposure: - Serum ATYR1940 concentrations: at week 1, every 6 weeks and at 1-week follow-up | — |
Countries
Denmark, Italy
Contacts
Public ContactClinical Trial Operations
Voisin Consulting
Outcome results
None listed