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

Phase 1

• Conditions: Muscular Atrophy, Spinal; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2020-003492-18-IT
• Lead Sponsor: BIOGEN IDEC RESEARCH LIMITED

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-08
• Last Update Posted: 17 August 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.
Are the trial subjects under 18? yes
Number of subjects for this age range:
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.

Study & Design

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