NCT01839656

A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 21 Days – 210 Days
• Healthy volunteers: Not accepted

Summary

The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.

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	Administered by intrathecal (IT) injection

Timeline

• Start date: 2013-05-08
• Primary completion: 2017-08-21
• Completion: 2017-08-21
• First posted: 2013-04-25
• Last updated: 2021-02-17
• Results posted: 2018-10-30

Locations

4 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study