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Antisense Oligonucleotide

Nusinersen for SMA (RESPOND Trial)

Phase 4
Waitlist Available
Research Sponsored by Biogen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Must have received IV onasemnogene abeparvovec after SMA symptom onset
SMN2 copy number of ≥1
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to day 778
Awards & highlights

RESPOND Trial Summary

This trial is evaluating nusinersen as a treatment for SMA in patients who have previously received onasemnogene abeparvovec. The objectives are to assess the safety and efficacy of nusinersen in this population.

Who is the study for?
This trial is for children under 36 months with Spinal Muscular Atrophy who have a specific genetic mutation and at least one copy of the SMN2 gene. They must have previously received onasemnogene abeparvovec, not been exposed to Nusinersen before, and can't be suffering from severe side effects related to onasemnogene abeparvovec.Check my eligibility
What is being tested?
The study tests the effectiveness of Nusinersen in young patients with SMA who've already been treated with onasemnogene abeparvovec. It aims to see if additional treatment improves clinical outcomes and assesses safety and tolerability.See study design
What are the potential side effects?
While specific side effects for this trial are not listed, common ones associated with Nusinersen include respiratory issues, constipation, headache, back pain, and potential post-lumbar puncture syndrome.

RESPOND Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I received IV onasemnogene abeparvovec after showing symptoms of SMA.
Select...
My genetic test shows I have at least one copy of the SMN2 gene.
Select...
I have received onasemnogene abeparvovec treatment before.
Select...
I am 3 years old or younger when I first received Nusinersen.
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I have a confirmed genetic form of SMA due to SMN1 changes.
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I received onasemnogene abeparvovec treatment over 2 months ago.
Select...
I received IV onasemnogene abeparvovec when I was 6 weeks old or younger.
Select...
I received IV onasemnogene abeparvovec between 43 and 180 days old.
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My SMA symptoms started before I was 4 months old.
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My child is less than 9 months old.
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My genetic test shows I have 2 copies of the SMN2 gene.
Select...
I have a confirmed genetic form of spinal muscular atrophy.

RESPOND Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day 778
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 778 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score
Secondary outcome measures
Change From Baseline in Cerebrospinal Fluid (CSF) Levels of Neurofilament Light Subunit (NF-L)
Change From Baseline in Plasma Levels of NF-L
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score
+8 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

RESPOND Trial Design

1Treatment groups
Experimental Treatment
Group I: Nusinersen 12 mgExperimental Treatment1 Intervention
Participants will receive Nusinersen 12 milligrams (mg) via intrathecal (IT) injection as loading doses on Days 1, 15, 29, and 64 followed by maintenance doses, every 4 months, on Days 183, 302, 421, 540 and 659.
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
467,388 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,769 Previous Clinical Trials
8,062,154 Total Patients Enrolled

Media Library

Nusinersen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04488133 — Phase 4
Spinal Muscular Atrophy Research Study Groups: Nusinersen 12 mg
Spinal Muscular Atrophy Clinical Trial 2023: Nusinersen Highlights & Side Effects. Trial Name: NCT04488133 — Phase 4
Nusinersen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04488133 — Phase 4

Frequently Asked Questions

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

Is this research novel in any way?

"Nusinersen has had a short but eventful research history. In 2015, the first study was conducted with 25 participants and sponsored by Biogen. This initial trial led to Nusinersen's Phase 2 drug approval. Currently, there are 7 active clinical trials taking place in 70 cities and 31 countries around the world."

Answered by AI

Do I fit the profile of somebody who could join this clinical trial?

"Up to 60 infants and young children, aged 2-36 months old, who have spinal muscular atrophy (SMA) may be enrolled in this study. In order to qualify, patients must also possess a genetic deletion or mutation of the survival motor neuron 1 (SMN1) gene consistent with 5q SMA homozygosity or compound heterozygosity. Additionally, participants must have received onasemnogene abeparvovec in the past and present suboptimal clinical status as decided by the study's principle Investigator."

Answered by AI

Are there any open vacancies for this trial?

"This clinical trial, as reflected on the website clinicaltrials.gov, is still enrolling patients. The trial was first posted on 1/7/2021 and most recently updated on 11/1/2022."

Answered by AI

Does this clinical trial have an age limit for participants?

"This research is focusing on children who are between 2 and 36 months old."

Answered by AI

Does Nusinersen put patients at risk for any adverse health effects?

"Nusinersen is classified as a Phase 4 treatment, meaning that it has been approved, and thus receives a safety score of 3."

Answered by AI

How many individuals are being experimented on in this clinical trial?

"Sixty individuals that meet the pre-set inclusion criteria are necessary to move forward with this research. Currently, patients at Stanford Neuromuscular Research in Palo Alto, California and Utah Program for Inherited Neuromuscular Disorders, University of Utah, Primary Children's Hospital in Salt Lake City, Utah can take part in the study."

Answered by AI

In how many different medical facilities is this research being conducted?

"To make things as convenient as possible for enrollees, the 12 sites for this clinical trial are situated in or near major cities like Palo Alto, Salt Lake City and Philadelphia. Other participating clinics are located in 12 other locations."

Answered by AI

Are there any similar drugs to Nusinersen that have been trialed before?

"Currently, there are 7 worldwide clinical trials involving Nusinersen. Out of these, 4 have reached Phase 3. Though several of the trials are based in Barcelona and Colorado, a total of 205 locations are running tests."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Stanford Neuromuscular Research
What portion of applicants met pre-screening criteria?
Met criteria
~15 spots leftby Oct 2025