Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1

Phase 1

• Conditions: Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1; MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2017-000266-29-IT
• Lead Sponsor: AVEXIS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-06-09
• Study Completion: 2020-09-11
• Last Update Posted: 12 July 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

• Patients with SMA Type 1 as determined by the diagnosis of SMA based on gene mutation analysis with biallelic SMN1 mutations (deletion or
point mutations) and one or two copies of SMN2 [inclusive of the known SMN2 gene modifier mutation (c.859G>C)]
• Patients must be < 6 months (< 180 days) of age at the time of AVXS-101 infusion.
• Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
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

Exclusion Criteria

• Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry < 95% saturation at screening
- Pulse oximetry saturation must not decrease = four (4) percentage points between screening and dosing with confirmatory oximetry reading
- 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.
• Use or requirement of non-invasive ventilatory support for 12 or more hours daily in the two weeks prior to dosing.
• Patient with signs of aspiration based on a swallowing test or whose weight-for-age falls below the 3rd percentile based on World Health Organization (WHO) Child Growth Standards [27] and is unwilling to use an alternative method to oral feeding.
• Participation in recent SMA treatment clinical study (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 (e.g., nusinersen, valproic acid) at any time prior to screening for this study. Oral ß- 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.
• Patient < 35 weeks gestational age at time of birth.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified