Overview

This trial is active, not recruiting.

Condition spinal muscular atrophy
Treatments ionis-smn rx, sham procedure
Phase phase 3
Sponsor Ionis Pharmaceuticals, Inc.
Start date July 2014
End date June 2017
Trial size 111 participants
Trial identifier NCT02193074, ISIS 396443-CS3B

Summary

This study will test the Clinical Efficacy and Safety of IONIS-SMN Rx in patients with infantile-onset Spinal Muscular Atrophy.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Administered by intrathecal injection
ionis-smn rx ISIS 396443
Administered by intrathecal injection
(Sham Comparator)
Small needle prick on the lower back at the location where the IT injection is normally made
sham procedure
Small needle prick on the lower back at the location where the IT injection is normally made

Primary Outcomes

Measure
Time to death or permanent ventilation
time frame: Up to 13 months
Proportion of motor milestone responders
time frame: Up to 13 months

Secondary Outcomes

Measure
Proportion of CHOP-INTEND responders
time frame: Up to 13 months
Proportion of CMAP responders
time frame: Up to 13 months
Percent of subjects not requiring permanent ventilation
time frame: Up to 13 months
Survival rate
time frame: Up to 13 months
Time to death or permanent ventilation in the subgroups of subjects below the study median disease duration
time frame: Up to 13 months
Time to death or permanent ventilation in the subgroups of subjects above the study median disease duration
time frame: Up to 13 months

Eligibility Criteria

Male or female participants up to 210 days old.

Inclusion Criteria: - Be 7 months (210 days) of age or younger at screening - Be born (gestational age) between 37 and 42 weeks - Be medically diagnosed with spinal muscular atrophy (SMA) - Have SMN2 Copy # of 2 - Body weight equal to or greater than 3rd percentile for age using appropriate country-specific guidelines - Be able to follow all study procedures - Reside within 9 hours ground travel each way, for the duration of the study - Meet additional study-related criteria Exclusion Criteria: - Hypoxemia (O2 saturation awake less than 96% or O2 saturation asleep less than 96%, without ventilation support) during screening evaluation - Meet additional study-related criteria

Additional Information

Official title A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy
Description This is a Phase 3 multicenter, double-blind, randomized, sham-procedure controlled study of IONIS-SMN Rx in patients with infantile-onset SMA. Subjects will be randomized 2:1 to receive IONIS-SMN Rx or a sham procedure control, respectively.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Ionis Pharmaceuticals, Inc..