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DEVOTE: Study of Nusinersen in Children with Spinal Muscular Atrophy

About this study

This was a research study to learn more about a drug to treat spinal muscular atrophy (SMA). SMA is a genetic disease that causes you to lose muscle control. There is no cure.

The drug is called nusinersen. It is also known as Spinraza. The U.S. Food and Drug Administration (FDA) has approved nusinersen as a treatment for SMA.

In this study, we wanted to find the best and safest dose of nusinersen to improve muscle function. We injected the drug into a participant's lower back using a thin needle. This procedure is called a lumbar puncture.

Eligibility overview

  • Genetic documentation of 5q SMA
  • Ages 1 week to adult (age varies by study part)

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Overview

Full title:

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy

Study goal:

The main goal of this study was to learn the best and safest dose of nusinersen that would improve muscle or motor function in patients with SMA.

Diagnosis:

Spinal muscular atrophy

Age:

Ages 1 week to adult

Clinical trials categories:

Neurological Disorders Spinal Muscular Atrophy (SMA)

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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