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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 belowSelect...
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:Select...
I have not had gene therapy, ASO treatment, or cell transplantation for SMA.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to day 2891
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day 2891
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 • NCT0229253745%
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not had gene therapy, ASO treatment, or cell transplantation for SMA.I have a confirmed genetic form of SMA.I show signs that my doctor thinks could be SMA.You have important problems with your blood or other body chemicals.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.I am 6 weeks old or younger.You meet other requirements specific to the study.Your oxygen levels are too low, even when you are resting or sleeping, and you don't use any extra oxygen or breathing support.You are carrying a baby for at least 37 weeks but no more than 42 weeks for one baby, and at least 34 weeks but no more than 42 weeks for two babies.
Research Study Groups:
This trial has the following groups:- Group 1: Nusinersen
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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