An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies

Phase 1

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

• Registration Number: EUCTR2015-001870-16-DE
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-10-28
• Study Completion: 2023-08-21
• Last Update Posted: 21 May 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 292

Inclusion Criteria

Subjects must meet all of the following criteria at Screening to be
eligible:
1. Signed informed consent of parent or guardian and signed informed assent of subject, if indicated per subject's age and institutional guidelines.
2. Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks.
Are the trial subjects under 18? yes
Number of subjects for this age range: 278
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 14
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Subjects meeting any of the following criteria are not eligible for the study:
- Have any condition or worsening of existing condition which in the opinion
of the Investigator would make the participant unsuitable for enrollment, or could
interfere with the participant participating in or completing the study
- Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogramm (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
- Participant’s parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
- Treatment with another investigational agent, biological agent, or device within
one month of Screening, or 5 half-lives of study agent, whichever is longer
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

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