Comparing ASP2957 with Methylprednisolone for x-linked myotubular myopathy

Exclusion

• −Participant has a known or suspected contraindication or hypersensitivity to methylprednisolone, prednisolone, sirolimus or any components of the ASP2957 formulation.
• −Active hepatitis A virus (HAV), hepatitis B virus (HBV) or hepatitis C virus (HCV)
• −Prior HBV or HCV virus infection due to the risk of reactivation associated with immunosuppression
• −human immunodeficiency virus type 1 (HIV-1) and human immunodeficiency virus type 2 (HIV-2)
• −Participant born \< 35 weeks gestation is still not term as per corrected age.
• −Participant is nutritionally unstable with weight less than fifth percentile for age or has a vitamin A, E or K deficiency.
• −Participant requires supplemental oxygen on a routine or chronic basis.
• −Note: The use of supplemental oxygen for acute, self-limited illnesses (for example, during hospitalization for pneumonia) shall not be exclusionary, provided the participant is neither acutely ill nor using supplemental oxygen at the time of screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
• −Participant currently has a clinically important respiratory infection or other clinically important active infection of any kind.
• −Participant has an active viral or bacterial infection including, but not limited to, positive testing for the following:
• −tuberculosis (TB) using the QuantiFERON-TB test
• −coronavirus disease 2019 (COVID-19)
• −cytomegalovirus (CMV), viral loads ≥ 500 IU/mL or attributable symptoms or evidence of end-organ disease due to CMV.
• −Participant has any history of cholestatic liver dysfunction and/or treatment for cholestasis. If the participant is taking prophylactic treatment for cholestasis (e.g., ursodiol, cholestyramine, rifampin or other therapies) which has not been prescribed for cholestatic liver dysfunction, treatment must be discontinued for at least 4 weeks prior to signing the ICFInformed consentThe process of being told what taking part involves, then choosing freely.Read more →.
• −Neonatal hyperbilirubinemia resolving within 4 weeks of birth in a full-term infant is not an exclusionExclusion criteriaThe things that would prevent someone from taking part.Read more →.
• −Participant has prior history of abnormal transaminases (alanine aminotransferase (ALT) or aspartate aminotransferase (AST)) and/or abnormal bilirubin metabolism associated with ascites, jaundice (aside from neonatal hyperbilirubinemia) or gastrointestinal bleeding.
• −Participant has a significant medical condition or life-threatening disease other than XLMTM that would interfere with adhering to protocolProtocolThe detailed plan a study must follow.Read more → requirements or would increase the risk of immunosuppression and/or recombinant adeno-associated virus (rAAV) administration.
• −Participant has musculoskeletal complications such as severe contractures and/or scoliosis that would limit the ability to observe improvements in neuromuscular function.
• −In participants with scoliosis, the Cobb angle must be \< 40 degrees for study eligibilityEligibility criteriaThe full list of requirements for taking part in a study.Read more →.
• −Other than as required per protocol, participant has received or plans to receive systemic immunomodulating agents within 90 days before day 1 (use of inhaled corticosteroids to manage chronic respiratory conditions is allowed).
• −Participant has previously received monoclonal antibodies of any type.
• −Exception: Monoclonal antibodies to prevent RSV are permitted, except for during the 4 weeks prior to the initiation of immunosuppression.
• −Exception: Standard of careStandard of careThe treatment normally given for a condition outside a study.Read more → surgical interventions such as gastrostomy, jejunostomy and Nissen fundoplication procedures are allowed.
• −Participant received any treatment for cholestasis (e.g., ursodiol, cholestyramine, rifampin or other therapies) prior to signing the ICF.
• −Participant is participating in another interventional studyInterventional studyA study where participants are given something to see what happens.Read more → or has received an adeno-associated virus (AAV)-based gene therapy.
• −Participant tests positive for anti-MyoAAV3.8 TAb, as determined by central laboratory testing.
• −Since very young children may have passive antibodies transferred in utero from the mother, participants ≤ 6 months of age who initially test positive for anti-MyoAAV3.8 total antibody (TAb) may be rescreened for study eligibility.
• −Participant has a contraindication to general anesthesia, magnetic resonance imaging (MRI) or muscle biopsy procedures.
• −Any other reason that would render the participant unsuitable for participation in the study, including risk of non-adherence to the study assessments and protocol.
• −Participant plans to have surgery within 12 weeks prior to day 1 through week 52 that may confound safety and efficacy data interpretation of the study intervention.