NCT02462759

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS 396443-CS4 (NCT02292537). The secondary objective of Part 1 of this study is to examine the pharmacokinetics (PK) of Nusinersen in participants with SMA. The primary objective of Part 2 of this study is to assess the long-term safety and tolerability of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments. The secondary objective of Part 2 of this study is to examine the PK of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments.

Detailed description

Part 2 is an Open Label extension phase.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Nusinersen	Administered by intrathecal injection.
PROCEDURE	Sham Procedure	Small needle prick on the lower back at the location where the IT injection is normally made.

Timeline

• Start date: 2015-08-19
• Primary completion: 2018-09-24
• Completion: 2018-09-24
• First posted: 2015-06-04
• Last updated: 2021-02-17
• Results posted: 2020-01-27

Locations

7 sites across 2 countries: United States, Germany

Regulatory

• FDA-regulated drug study