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Antisense Oligonucleotide

Nusinersen for Spinal Muscular Atrophy (ONWARD Trial)

Phase 3
Waitlist Available
Research Sponsored by Biogen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Key
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up today 1921
Awards & highlights

ONWARD Trial Summary

This trial is for participants with Spinal Muscular Atrophy who have previously participated in another study. They will be given nusinersen, which is a drug meant to help with the disease. The study is to see if the drug is safe and effective long-term.

Who is the study for?
This trial is for individuals with spinal muscular atrophy (SMA) who completed the Day 302 visit in a prior study (NCT04089566). They must not have received other investigational therapies or participated in another interventional clinical study, nor started any approved SMA therapy after that specific visit.Check my eligibility
What is being tested?
The trial is testing the long-term safety and effectiveness of higher doses of Nusinersen given through spinal injection to those with SMA. Participants are from a previous Nusinersen study and will continue to receive this drug to assess ongoing benefits and risks.See study design
What are the potential side effects?
Potential side effects include back pain, headache, vomiting, and potential risk of infection at the injection site. There may also be complications related to the nervous system due to intrathecal administration.

ONWARD Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
Please provide more details for an accurate summary.

ONWARD Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up today 1921
This trial's timeline: 3 weeks for screening, Varies for treatment, and up today 1921 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from Baseline in Activated Partial Thromboplastin Time (aPTT)
Change from Baseline in Growth Parameters
Change from Baseline in International Normalized Ratio (INR)
+10 more
Secondary outcome measures
Change From Baseline in Plasma Levels of Neurofilament (NF)
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
+10 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

ONWARD Trial Design

2Treatment groups
Experimental Treatment
Group I: BIIB058 50/28 mg (Prior Maintenance Dose 12 mg)Experimental Treatment1 Intervention
Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.
Group II: BIIB058 28 mg (Prior Maintenance Dose 28 mg)Experimental Treatment1 Intervention
Participants who received maintenance dose of 28 milligrams (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921.
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
638 Previous Clinical Trials
467,281 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,766 Previous Clinical Trials
8,060,648 Total Patients Enrolled

Media Library

Nusinersen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04729907 — Phase 3
Spinal Muscular Atrophy Research Study Groups: BIIB058 28 mg (Prior Maintenance Dose 28 mg), BIIB058 50/28 mg (Prior Maintenance Dose 12 mg)
Spinal Muscular Atrophy Clinical Trial 2023: Nusinersen Highlights & Side Effects. Trial Name: NCT04729907 — Phase 3
Nusinersen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04729907 — Phase 3

Frequently Asked Questions

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

Where can patients find this trial?

"Currently, this clinical trial is being run out of 7 sites. The locations include London, Baltimore, Sacramento, and 7 other sites. To minimize travel demands, patients are encouraged to enroll at the site nearest them."

Answered by AI

Does this experiment still have room for more test subjects?

"This clinical trial is not presently looking for new participants. The trial was first posted on 4/19/2021 and was most recently updated on 10/21/2022. If you are exploring other studies, there are presently 205 studies actively admitting patients with muscular atrophy, spinal and 7 studies for Nusinersen actively enrolling participants."

Answered by AI

Is Nusinersen a new medical treatment?

"There are 7 ongoing studies involving Nusinersen, 4 of which are in the critical Phase 3. The largest number of research centres are based in Madrid and Washington, but there are a total of 205 locations running trials for this medication."

Answered by AI

Will patients experience any harmful side-effects from Nusinersen?

"Nusinersen has received a safety rating of 3 from our team at Power. This is because Nusinersen is in Phase 3 clinical trials, which suggests that there is both efficacy and safety data available."

Answered by AI

What is the significance of this clinical trial in the medical world?

"Nusinersen has been under clinical investigation since 2015 when Biogen sponsored its first study. After the Phase 1 clinical trial with 25 patients, Nusinersen was approved for Phase 2 drug trials. To date, there are 7 active Nusinersen trials being conducted in 31 different countries and across 70 cities."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Research Site
What portion of applicants met pre-screening criteria?
Did not meet criteria
~64 spots leftby Jul 2026