CTIS2024-512637-32-00招募中1 期
ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ATX-MTM-002
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 26
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- Male
入选标准
- •1. Subject has a diagnosis of XLMTM resulting from a genetically confirmed mutation in the MTM1 gene as assessed by a Sponsor- approved testing facility.* * = Inclusion/exclusion criteria for delayed treatment control subjects before receiving AT132., 2. Subject is male.*, 3. Subject is aged less than 5 years old at dosing*, 4. Subject requires mechanical ventilatory support: Part 1: Subject requires some mechanical ventilatory support (eg., ranging from 24 hours per day full time mechanical ventilation, to noninvasive support such as continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP] during sleeping hours). Part 2: Subject requires invasive mechanical ventilatory support ranging from 20 to 24 hours per day at screening (confirmed by daytime polysomnographic study)., 5. Subject requiring invasive mechanical ventilator support is fitted with or willing to be fitted with a cuffed tracheostomy tube for some respiratory assessments.*, 6. Subject has ventilator maximum positive end-expiratory pressure (PEEP) < 8 cm H2O at screening.*, 7. Signed informed consent by the parent(s) or legally authorized representative(s) (LAR) (when applicable).*, 8. Subject and parent(s)/LAR(s) are willing and able to comply with study visits and study procedures.*, 9. Subject's range is = 4.8 kg (UNIQUE to France)
排除标准
- •1. Subject is participating in an interventional study designed to treat XLMTM.* * = If a subject is a delayed-treatment control, this inclusion/exclusion criterion must be met before receiving AT132., 10.Subject has a contraindication to prednisolone.*, 11.Subject has a contraindication to study drug or ingredients.*, 12. Subject has previous scoliosis repair surgery/procedure, or planned/expected scoliosis repair surgery/procedure, in the 12 months following Day 1 (Part 2 including any subjects enrolles under protocol v8 and beyond )., 13. Subject has contractures, scoliosis, or other medical condition that would limit the potential to achieve unassisted sitting, in the opinion of the Investigator (Part 2 including any subjects enrolles under protocol v8 and beyond )., 14. Subject is able to sit without assistance for at least 30 seconds at screening, in the opinion of the Investigator (Part 2 including any subjects enrolles under protocol v8 and beyond )., 15. Subject has a clinically important condition, including CTCAE v4.03 Grade = 2 anemia (< 10 g/dL hemoglobin).*, 16. Subject has a contraindication to ursodiol (ursodeoxycholic acid).*, 17. Subject has a prior diagnosis or history of cardiac arrhytmias, myocarditis, or any other cardiac disease (UNIQUE to FRANCE)., 18. Subject has a contradiction to general anesthesia and to muscle biopsy procedure (UNIQUE to France)., 2. Subject born < 35 weeks gestation who is still not term as per corrected age., 3. Subject tests positive for AAV8 neutralizing antibody with titers > 1:20 (subjects under the age of 18 months may be retested in cases where antibodies may have been maternally acquired and titers may decline in the first months of life).*, 4. Subject had recent surgery (< 3 months before Day 1) or has planned surgery that may confound data collection during the first 48 weeks of the study., 5. Subject has a clinically important condition or life-threatening disease, other than XLMTM, in the opinion of the Investigator.*, 6. Subject has a clinically significant underlying liver disease, defined as: • = Grade 3 aspartate aminotransferase (AST) (> 5.0 x upper limit of normal [ULN]; Common Terminology Criteria for Adverse Events [CTCAE] v. 4.03)* • = Grade 3 alanine aminotransferase (ALT) (> 5.0 x ULN; CTCAE v. 4.03)* • Hepatic peliosis or any other clinically significant structural abnormality detected by ultrasound*, 7. Subject is currently experiencing a clinically important respiratory infection or other active infection.*, 8.Subject has received pyridostigmine or any medication to treat XLMTM within 3 months before Day 1.*, 9.Other than as required per protocol, subject has received immunemodulating agents within 3 months before Day 1 (use of inhaled corticosteroids to manage chronic respiratory conditions is allowed); use of other concomitant medications to manage chronic conditions must have beenstable for at least 4 weeks before dosing.*
研究者
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