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

NCT01839656 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2021-02-17

Study results available
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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.

Conditions

Interventions

DRUG

nusinersen

Administered by intrathecal (IT) injection

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
21 Days
Max Age
210 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-05-08
Primary Completion
2017-08-21
Completion
2017-08-21
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01839656 on ClinicalTrials.gov