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Nusinersen for SMA (RESPOND Trial)
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.
RESPOND Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowRESPOND Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2017 Phase 3 trial • 126 Patients • NCT02292537RESPOND Trial Design
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Who is running the clinical trial?
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- Your overall health condition is not good enough according to the doctor in charge.I received IV onasemnogene abeparvovec after showing symptoms of SMA.My genetic test shows I have at least one copy of the SMN2 gene.I have received onasemnogene abeparvovec treatment before.I am 3 years old or younger when I first received Nusinersen.I am experiencing severe side effects from onasemnogene abeparvovec treatment.I have a confirmed genetic form of SMA due to SMN1 changes.I received onasemnogene abeparvovec treatment over 2 months ago.I received IV onasemnogene abeparvovec when I was 6 weeks old or younger.I haven't used any experimental drugs or treatments for SMA, except onasemnogene abeparvovec.I received IV onasemnogene abeparvovec between 43 and 180 days old.My SMA symptoms started before I was 4 months old.My child's weight is below the third percentile for their age, according to WHO standards.I have previously been treated with Nusinersen.My child is less than 9 months old.My genetic test shows I have 2 copies of the SMN2 gene.I have a confirmed genetic form of spinal muscular atrophy.
- Group 1: Nusinersen 12 mg
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- 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.
Frequently Asked Questions
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."
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."
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."
Does this clinical trial have an age limit for participants?
"This research is focusing on children who are between 2 and 36 months old."
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."
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."
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."
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."
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What portion of applicants met pre-screening criteria?
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