Home
Clinical Trials
A Study for Participants With...

A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Last Updated: 2024-10-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

292 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-11-04

Study Completion Date

2023-08-21

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

NCT02292537

Spinal Muscular Atrophy

COMPLETED PHASE3

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

NCT02462759

Spinal Muscular Atrophy

TERMINATED PHASE2

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

NCT02052791

Spinal Muscular Atrophy

COMPLETED PHASE1

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

NCT01494701

Spinal Muscular Atrophy

COMPLETED PHASE1

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

NCT02193074

Spinal Muscular Atrophy

TERMINATED PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This study was initiated and the protocol was registered by Ionis Pharmaceuticals, Inc.

In August 2016, Biogen assumed responsibility for this study.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Spinal Muscular Atrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

During the blinded loading period, the following participants will be masked:

* Key site personnel (Investigator, Study Coordinator, and Outcomes Assessors)
* Participant
* The sponsor

After the loading period has been completed, subsequent doses will be unblinded.

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Group 1

Participants transitioned from ISIS 396443-CS3B (NCT02193074)

Group Type EXPERIMENTAL