2025-523213-29-00招募中2 期
A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants with Invasive Ventilator-dependent X-linked Myotubular Myopathy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Incidence and severity of TEAEs and AESIs
研究概览
简要总结
To evaluate the safety and tolerability of ASP2957 and to determine the recommended dose level
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Participant is projected to be ≤ 36 months of age at dosing
- •Participant has molecular genetic report from a CAP-approved testing facility at screening that confirms a diagnosis of XLMTM and harbors a “pathogenic” or “likely pathogenic” variant in the MTM1 gene as classified using the ACMG standards and guidelines for interpretation of sequence variants
- •Participant is ventilator-dependent and meets the following criteria: a. Required respiratory support at birth b. Requires ≥ 20 hours per day of invasive ventilator support (confirmed during screening) c. Has a tracheostomy tube
- •Participant has no evidence of hepatic peliosis, increased echogenicity or any other clinically important abnormal finding on liver ultrasound as assessed by the investigator in consultation with the site hepatologist
- •Participant’s hepatobiliary laboratory measurements must meet the following criteria during screening and for the 2-month retrospective assessment of participant’s medical history: a. a. ALT and AST < 3 × ULN b. Direct bilirubin ≤ 1 × ULN c. Serum total bile acids < 2 × ULN, independent of fasting status
- •Participant’s parent(s) or LAR(s) must provide documentation of being current with recommended immunization schedule according to regional guidelines.
排除标准
- •Participant born < 35 weeks gestation is still not term as per corrected age.
- •Participant tests positive for anti-MyoAAV3.8 TAb, as determined by central laboratory testing. a. Note: 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 TAb may be rescreened for study eligibility.
- •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. a. 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 screening.
- •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: a. TB using the QuantiFERON-TB test b. Active HAV, HBV or HCV c. Prior HBV or HCV virus infection due to the risk of reactivation associated with immunosuppression d. HIV-1 and HIV-2 e. COVID-19 f. CMV, including either positive CMV IgG or presence of detectable CMV DNA by quantitative PCR at screening.
- •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 ICF. a. Neonatal hyperbilirubinemia resolving within 4 weeks of birth in a full-term infant is not an exclusion.
- •Participant has prior history of abnormal transaminases (ALT or AST) and/or abnormal bilirubin metabolism associated with ascites, jaundice (aside from neonatal hyperbilirubinemia) or gastrointestinal bleeding.
- •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 received any treatment for cholestasis (e.g., ursodiol, cholestyramine, rifampin or other therapies) prior to signing the ICF.
结局指标
主要结局
Incidence and severity of TEAEs and AESIs
Incidence and severity of TEAEs and AESIs
Change from baseline in the following safety assessments through week 52: o clinical laboratory tests o cardiac findings from ECG and ECHO o muscle findings from muscle MRI and histopathology o physical examinations
Change from baseline in the following safety assessments through week 52: o clinical laboratory tests o cardiac findings from ECG and ECHO o muscle findings from muscle MRI and histopathology o physical examinations
次要结局
- Change from baseline in hours per day of ventilation support at week 52
- ASP2957 vector DNA in serum through week 52
- ASP2957 vector DNA in muscle biopsy at week 52
- ASP2957 vector DNA in saliva, urine and stool
- Immunogenicity of ASP2957 through week 52 as assessed with the following tests: o Anti-MyoAAV3.8 TAb and NAb o Anti-myotubularin Tab
研究者
RA Clinical Trial Unit Head
Scientific
Astellas Gene Therapies Inc.
研究点 (1)
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