NCT01703988

An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 2 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.

Detailed description

This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, sponsorship of the trial was transferred to Biogen.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Nusinersen	Single IT injection for each dose

Timeline

• Start date: 2012-10-31
• Primary completion: 2015-01-31
• Completion: 2015-01-31
• First posted: 2012-10-11
• Last updated: 2021-04-13
• Results posted: 2017-03-13

Locations

4 sites across 1 country: United States