Nusinersen for Spinal Muscular Atrophy

(NURTURE Trial)

Recruiting at 40 trial locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Biogen

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

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 (Antisense Oligonucleotide)

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

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