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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 to day 1921
Awards & highlights
No Placebo-Only Group
Pivotal 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.
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.
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.

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.

Timeline

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from Baseline in Growth Parameters
Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
+1 more
Secondary study objectives
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
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones
+4 more

Side effects data

From 2017 Phase 3 trial • 126 Patients • NCT02292537
45%
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.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
~340

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor
646 Previous Clinical Trials
467,398 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,887 Previous Clinical Trials
8,088,917 Total Patients Enrolled

Media Library

Nusinersen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04729907 — Phase 3
Spinal Muscular Atrophy Research Study Groups: BIIB058 50/28 mg (Prior Maintenance Dose 12 mg), BIIB058 28 mg (Prior Maintenance Dose 28 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
~37 spots leftby Jul 2026
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