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Antisense Oligonucleotide

Nusinersen for Spinal Muscular Atrophy (NURTURE Trial)

Phase 2
Waitlist Available
Research Sponsored by Biogen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation
Genetic documentation of 2 or 3 copies of survival motor neuron 2 (SMN2)
Must not have
Treatment with an investigational drug given for the treatment of SMA biological agent, or device. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to day 2891
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing if a certain medication can help prevent or delay the need for respiratory intervention or death in infants with a genetic diagnosis of Spinal Muscular Dystrophy.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions at the injection site, back pain, headache, fever, post-lumbar puncture syndrome and potential risks associated with intrathecal administration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a confirmed genetic form of SMA.
Select...
My genetic test shows I have 2 or 3 copies of the SMN2 gene.
Select...
I am 6 weeks old or younger.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have not had gene therapy, ASO treatment, or cell transplantation for SMA.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day 2891
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 2891 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Time to death or respiratory intervention
Secondary study objectives
Change from Baseline in clinical laboratory parameters
Change from Baseline in vital signs

Side effects data

From 2017 Phase 3 trial • 126 Patients • NCT02292537
45%
Upper respiratory tract infection
36%
Pyrexia
36%
Nasopharyngitis
21%
Cough
19%
Gastroenteritis
17%
Rhinorrhoea
17%
Joint contracture
14%
Pneumonia
12%
Vomiting
12%
Ear infection
10%
Constipation
10%
Bronchitis
10%
Gastroenteritis viral
10%
Otitis media
10%
Arthralgia
7%
Diarrhoea
7%
Pharyngitis streptococcal
7%
Pain in extremity
7%
Scoliosis
7%
Headache
5%
Faecaloma
5%
Influenza
5%
Dehydration
5%
Respiratory distress
5%
Conjunctivitis
5%
Upper respiratory tract congestion
2%
Pneumonia adenoviral
2%
Respiratory syncytial virus infection
2%
Respiratory failure
2%
Pneumonia parainfluenzae viral
100%
80%
60%
40%
20%
0%
Study treatment Arm
Sham Procedure
Nusinersen

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: NusinersenExperimental Treatment1 Intervention
Nusinersen administered as an intrathecal injection
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Nusinersen
2014
Completed Phase 3
~340

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor
646 Previous Clinical Trials
467,488 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,885 Previous Clinical Trials
8,088,760 Total Patients Enrolled

Media Library

Nusinersen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT02386553 — Phase 2
Spinal Muscular Atrophy Research Study Groups: Nusinersen
Spinal Muscular Atrophy Clinical Trial 2023: Nusinersen Highlights & Side Effects. Trial Name: NCT02386553 — Phase 2
Nusinersen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02386553 — Phase 2
~0 spots leftby Dec 2024