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DEVOTE: Study of Nusinersen in Children with Spinal Muscular Atrophy (CLOSED TO ACCRUAL)

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


Neurological Diseases

Diseases Treated:

Spinal muscular atrophy

Eligibility Overview:

  1. Brief Summary

    This is study to evaluate nusinersen (Spinraza) in participants with infantile-onset and later-onset spinal muscular atrophy (SMA).

    Nusinersen is an antisense oligonucleotide (ASO) designed to increase SMN protein expression and improve motor function. It is approved in the U.S. and other countries for treatment of children and adults with SMA at a dosage of 12 mg administered intrathecally at specified intervals.

    This study investigates the efficacy, safety, and tolerability of a higher-dose nusinersen regimen consisting of a 50 mg loading dose and 28 mg maintenance dose to achieve rapid drug exposure. Data from preclinical studies suggest dosage levels above 28 mg may enhance motor function response.

    Part A of the study enrolled children and adolescents with later-onset SMA and is completed. Part B  is currently enrolling participants with infantile-onset SMA. Part C is enrolling a limited number of participants of all ages with SMA who have been given nusinersen for at least one year prior to the study.

    Primary Objective

    • To evaluate the efficacy of nusinersen administered intrathecally at higher doses in patients with SMA

    Eligibility Criteria

    Key Inclusion Criteria include:

    Part A – This part is completed

    Part B

    • Genetic documentation of 5q SMA
    • Infantile-onset SMA, 1 week to 7 months old
    • Later-onset SMA, at least 2 years old and younger than 10 years old (cohort filled, no longer enrolling)

    Part C – This part has completed enrollment.

    • Genetic documentation of 5q SMA
    • At least 18 years old
    • Received treatment with nusinersen for at least 1 year prior to study

    Key Exclusion Criteria include:

    • Active infection requiring systemic antiviral or antimicrobial therapy
    • History of brain or spinal cord disease that would interfere with study procedures
    • History of bacterial meningitis, viral encephalitis or hydrocephalus
    • Treatment with an investigational drug given for treatment of SMA, biological agent or device within 30 days or 5 half-lives of the agent
    • Prior ASO treatment or cell transplantation
    • Prior or current treatment with any SMN2-splicing modifier or gene therapy
    • Pregnant or breastfeeding

    Study Sites

    St. Jude Children’s Research Hospital
    Memphis, Tennessee

    Collaborating sites in and outside the U.S.

  2. About this study

    This is 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 want to find out the best and safest dose of nusinersen to improve muscle function. We will inject the drug into your lower back using a thin needle. This procedure is called a lumbar puncture.

    You may not benefit if you take part in this research study. However, you may help researchers decide the safest and most effective dose of nusinersen for patients with SMA.

    Purpose of this clinical trial

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

    Eligibility overview

    • Genetic documentation of 5q SMA
    • Ages 1 week to adult (age varies by study part)
    Sponsors: Biogen identifier NCT04089566
    Trial Start Date: March 2020     
    Estimated Enrollment: 172 globally (3-5 at St. Jude)
    Study Type: Interventional
    Study Phase: Phase 2/3  
    Conditions: Spinal muscular atrophy         
    Ages: 1 week to adult
    Principal investigator: Richard Finkel, MD
    Study Sites: St. Jude Children’s Research Hospital and collaborating sites outside the U.S.
    For a consultation or to discuss MNEMONIC: St. Jude Physician/Patient Referral Office

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.