Nusinersen for Spinal Muscular Atrophy

(NURTURE Trial)

Recruiting at 40 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Biogen

Must not be taking: Investigational drugs

Disqualifiers: Hypoxemia, Clinical SMA signs, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

25 Participants Needed

Trial Summary

What is the purpose of this trial?

The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.

Research Team

Medical Director

Principal Investigator

Biogen

Eligibility Criteria

This trial is for infants aged ≤ 6 weeks with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). They must have a specific genetic makeup of SMA, an ulnar nerve test result ≥ 1 mV, and be born within certain gestational age ranges. Infants cannot participate if they've had previous treatments for SMA or show signs of low oxygen levels or abnormal blood tests.

Inclusion Criteria

I have a confirmed genetic form of SMA.

My genetic test shows I have 2 or 3 copies of the SMN2 gene.

Your ulnar nerve's electrical activity is normal at the start of the study.

Exclusion Criteria

I have not had gene therapy, ASO treatment, or cell transplantation for SMA.

I show signs that my doctor thinks could be SMA.

You have important problems with your blood or other body chemicals.

Treatment Details

Interventions

• Nusinersen

Trial Overview The study is testing the effectiveness of multiple doses of Nusinersen given through spinal injection in preventing or delaying respiratory intervention or death in infants with SMA. It aims to understand how this treatment can affect these children before symptoms appear.

Participant Groups

Group I: Nusinersen Experimental Treatment 1 Intervention

Nusinersen administered as an intrathecal injection

Nusinersen is already approved in United States, European Union for the following indications:

Approved in United States as Spinraza for:

• Spinal muscular atrophy in pediatric and adult patients

Approved in European Union as Spinraza for:

• Spinal muscular atrophy

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada