← Back to Search

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)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 13 and 24 months of age
Awards & highlights

NURTURE Trial 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.Check my eligibility
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.See study design
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.

NURTURE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
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.

NURTURE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 13 months, and 2, 3, 4, 5, 6, 7 and 8 years of age
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 13 months, and 2, 3, 4, 5, 6, 7 and 8 years of age for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Time to death or respiratory intervention
Secondary outcome measures
Cerebrospinal fluid (CSF) and plasma Nusinersen concentrations
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
Change from Baseline in arm circumference ratio
+14 more

Side effects data

From 2017 Phase 3 trial • 126 Patients • NCT02292537
43%
Pyrexia
30%
Upper respiratory tract infection
29%
Vomiting
29%
Headache
25%
Back pain
25%
Cough
24%
Nasopharyngitis
11%
Gastroenteritis
10%
Diarrhoea
10%
Gastroenteritis viral
10%
Influenza
8%
Bronchitis
8%
Ear infection
7%
Rhinorrhoea
7%
Conjunctivitis
7%
Epistaxis
6%
Upper respiratory tract congestion
6%
Otitis media
6%
Constipation
5%
Pharyngitis streptococcal
5%
Pneumonia
5%
Arthralgia
5%
Pain in extremity
5%
Joint contracture
4%
Pneumonia viral
4%
Scoliosis
2%
Respiratory distress
1%
Post lumbar puncture syndrome
1%
Abdominal distension
1%
Pain
1%
Bacteraemia
1%
Parainfluenzae virus infection
1%
Pneumonia aspiration
1%
Respiratory syncytial virus bronchitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Nusinersen
Sham Procedure

NURTURE 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
~200

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor
639 Previous Clinical Trials
467,409 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,769 Previous Clinical Trials
8,062,175 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has recruitment for this investigation commenced?

"Unfortunately, according to clinicaltrials.gov the trial is not enrolling patients at this time; however, it was initially posted on May 20th 2015 and last updated November 11 2021. Despite this setback, there are over two hundred other trials that are currently recruiting participants."

Answered by AI

What is the participant quota for this experiment?

"This research project is no longer enrolling, as the clinical trial was first posted on May 20th 2015 and last revised on November 11th 2021. For those searching for other studies, there are 205 atrophic trials presently recruiting participants, in addition to 7 medical investigations seeking patients receiving Nusinersen treatments."

Answered by AI

How many medical facilities are actively conducting this trial?

"The current medical trial is enrolling participants at 8 distinct sites, located in Boston, New york and Los Angeles as well other locations. To reduce travel strain, one should consider the site closest to them if they wish to partake."

Answered by AI

Have any other trials been conducted utilizing Nusinersen?

"Nusinersen was initially studied in 2015 at Research Site and currently, there are 9 completed trials. There is also a notable number of ongoing medical studies that are being conducted from multiple sites within Boston, Massachusetts."

Answered by AI

What impact has Nusinersen had on patient safety metrics?

"With a score of 2, there has been some evidence collected on Nusinersen's safety profile, although the efficacy of this intervention remains unproven."

Answered by AI

Is this a pioneering endeavor with no precedent?

"Since its inception in 2015, Nusinersen has been rigorously explored. Biogen sponsored the initial trial involving 25 patients and it was shortly thereafter that Phase 2 clinical approval was granted. At present, 7 live trials are located across 70 cities and 31 countries."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What portion of applicants met pre-screening criteria?
Met criteria
~2 spots leftby Jan 2025