A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

Phase 1

• Conditions: Muscular Atrophy, Spinal; MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2020-003492-18-ES
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-09-01
• Last Update Posted: 7 September 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

- Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote).
- Must have previously received onasemnogene abeparvovec.
- Participants with suboptimal clinical status per the Investigator.

Note: other protocol defined Inclusion/Exclusion criteria may apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
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

- Prior exposure to nusinersen.
- Ongoing severe or serious AEs related to onasemnogene abeparvovec.
- Use of steroids within 1 month prior to first dose of nusinersen.

Note: other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

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