Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD) (FSHD)
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
About this trial
This is an interventional treatment trial for Limb-Girdle Muscular Dystrophies focused on measuring LGMD2B, FSHD
Eligibility Criteria
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.
Sites / Locations
- University of California, Irvine, ALS and Neuromuscular Center
- Kennedy Krieger Institute; The Johns Hopkins University School of Medicine
- OSU Wexner Medical Center
- Rigshospitalet, University of Copenhagen
- Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET)
- Institut de Myologie, Hôpital Pitié-Salpêtrière
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Group A - FSHD
Group B - LGMD2B and FSHD
Participants with FSHD will receive single dose of placebo intravenous (IV) infusion followed by ATYR1940 IV infusion at doses of 0.3 up to 1.0 milligrams/kilograms (mg/kg) once weekly for 8 weeks and then twice weekly for 4 weeks using intraparticipant dose escalation for up to 12 weeks.
Participants with LGMD2B and FSHD will receive single dose of placebo IV infusion followed by ATYR1940 IV infusion at doses of 0.3 up to 1.0 and 3.0 mg/kg once weekly for 8 weeks and then twice weekly for 4 weeks using intraparticipant dose escalation for up to 12 weeks.