Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion - AVXS-101

AveXis0 个研究点目标入组 5 人开始时间: 待定最近更新: 2024年4月15日

试验速览

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•1. Patients with SMA Type 1 as determined by the diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and one or two copies of SMN2 [inclusive of the known SMN2 gene modifier mutation (c.859G>C)]
•2. Patients must be < 6 months (< 180 days) of age at the time of AVXS 101 infusion.
•3. Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy.

•1. Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry < 95% saturation at screening
•a. Pulse oximetry saturation must not decrease * four ( 4) percentage points between screening and dosing with confirmatory oximetry reading
•b. Patients may be put on non-invasive ventilatory support for less than < 12 hours per day at the discretion of their physician or trial staff.;2. Use or requirement of non-invasive ventilatory support for *12 or more hours daily in the two 2 weeks prior to dosing.;3 Patient with signs of aspiration based on a swallowing test or whose weight-for-age is below the 3rd percentile based on World Health Organization (WHO) Child Growth Standards and is unwilling to use an alternative method to oral feeding.;4. Participation in recent SMA treatment clinical trial (with the exception of observational cohort studies or non-interventional studies) or receipt of an investigational or commercial compound, product or therapy administered with the intent to treat SMA (eg, nusinersen, valproic acid,) at any time prior to screening for this trial. Oral *-agonists must be discontinued at least 30 days before gene therapy dosing. Inhaled albuterol specifically prescribed for the purposes of respiratory (bronchodilator) management is acceptable.;5. Patient < 35 weeks gestational age at time of birth

发起方

AveXis