ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO

Audentes Therapeutics Inc.0 个研究点目标入组 12 人开始时间: 2018年5月25日最近更新: 2024年5月21日

适应症

试验速览

暂无简介。

•- Subject has a diagnosis of XLMTM resulting from a genetically confirmed mutation in the MTM1 gene as assessed by a Sponsor-approved testing facility.
•- Subject is male.
•- Subject is aged less than 5 years old at Day 1 and/or participated in the ATX-MTM-009 (INCEPTUS) study.
•- Subject's weight is = 4.8 Kg
•- Subject requires some mechanical ventilator 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).
•- Subject requiring invasive mechanical ventilator support is fitted with or willing to be fitted with a cuffed tracheostomy tube for some respiratory assessments.
•- Subject has ventilator maximum positive end-expiratory pressure (PEEP) <8 cm H2O at baseline.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 12
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•- Subject is participating in an interventional study designed to treat XLMTM.
•- Subject born <35 weeks gestation who is still not term as per corrected age.
•- Subject tests positive for AAV8 neutralizing antibody with titers above protocol specified threshold.
•- 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.
•- Subject has a clinically important condition other than XLMTM in the opinion of the investigator.
•- Subject has a clinically significant underlying liver disease.
•- Subject is currently experiencing a clinically important respiratory infection or other active infection.
•- Subject has received pyridostigmine or any medication to treat XLMTM within 3 months before Day 1.
•- Other than as required per protocol, subject has received immune-modulating 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 been stable for at least 4 weeks before dosing.

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