CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 21 enrolled
Drug / intervention
nusinersendrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01839656
NCT01839656Phase 2Completed
CompareCT.gov

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

Biogen·interventional·Posted Apr 25, 2013·Updated Feb 17, 2021

In Brief

A Phase 2 clinical trial evaluating nusinersen for Spinal Muscular Atrophy. Completed, enrolled 21 participants across 4 sites in 2 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesCanada, United States
Collaborators--

Timeline

Phase 2CompletedFinished
20132014201520162017201820192020202120222023202420252026
First PostedApr 25, 2013
Enrollment StartMay 8, 2013
Primary CompletionAug 21, 2017
TodayJul 2, 2026
Enrollment to primary: 4.3 yearsPosted 13.2 years ago

Interventions

nusinersendrug

Administered by intrathecal (IT) injection