At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 34 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.
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
In Brief
A Phase 2 clinical trial evaluating Nusinersen for Spinal Muscular Atrophy. Completed, enrolled 34 participants across 4 sites.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
20132014201520162017201820192020202120222023202420252026
First PostedOct 2012
Enrollment StartOct 2012
Primary CompletionJan 2015
TodayJul 2026
First PostedOct 11, 2012
Enrollment StartOct 31, 2012
Primary CompletionJan 31, 2015
TodayJul 2, 2026
Enrollment to primary: 2.3 yearsPosted 13.7 years ago
Interventions
Nusinersendrug
Single IT injection for each dose