ot applicable

Phase 1

• Conditions: Spinal Muscular Atrophy; 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: EUCTR2019-002611-26-IT
• Lead Sponsor: AVEXIS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-03
• Last Update Posted: 30 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 308

Inclusion Criteria

> Patients with SMA (with 1, 2 or 3copies of SMN2) who received AVXS-101 gene replacement therapy in an AveXis clinical study
> Patient/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 64
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

> Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study

Study & Design

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