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Gene Therapy

OAV101 for Spinal Muscular Atrophy (STEER Trial)

Phase 3
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnostic confirmation during screening period of 5q SMA
Able to sit independently at screening, but has never had the ability to walk independently.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to 52 weeks
Awards & highlights

STEER Trial Summary

This trial is testing a new treatment for people with a severe form of spinal muscular atrophy. The treatment will be given directly into the spinal fluid and will be evaluated for safety, effectiveness, and side effects over 15 months.

Who is the study for?
This trial is for children aged 2 to <18 with Type 2 spinal muscular atrophy (SMA) who can sit but not walk independently, have had symptoms since ≥6 months old, and haven't used any SMN-targeting therapies. They must pass a motor function test and not have severe contractures, liver issues, recent or planned spine/hip surgery, sensory abnormalities, high AAV9 antibody levels, recent infections or require extensive ventilation.Check my eligibility
What is being tested?
The trial tests the effectiveness of OAV101 given through the spine compared to a sham control in kids with SMA over 15 months. It aims to see if this treatment improves their condition without causing harm.See study design
What are the potential side effects?
While specific side effects are not listed here, intrathecal treatments like OAV101 may cause back pain at the injection site, headache, nausea or potential immune reactions due to its delivery method and genetic therapy nature.

STEER Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My condition is confirmed to be 5q spinal muscular atrophy.
Select...
I can sit by myself but have never been able to walk on my own.
Select...
I have never taken SMN-targeting therapies like Evrysdi or Spinraza.
Select...
I have never taken SMN-targeting therapies like Evrysdi or Spinraza.

STEER Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to 52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 52 weeks 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 Hammersmith Functional Motor Scale - Expanded (HFMSE) total score at the end of Follow-up Period 1 in treated patients compared to sham controls in the ≥ 2 to < 18 years age group
Secondary outcome measures
Achievement of at least a 3-point improvement from baseline in HFMSE total score at the end of Follow-up Period 1 in the ≥ 2 to < 18 years age group
Achievement of at least a 3-point improvement from baseline in HFMSE total score at the end of Follow-up Period 1 in the ≥ 2 to < 5 years age group
Change from baseline in HFMSE total score at the end of Follow-up Period 1 in treated patients compared to sham controls in the ≥ 2 to < 5 years age group
+6 more

Side effects data

From 2023 Phase 3 trial • 24 Patients • NCT04851873
71%
Vomiting
57%
Upper respiratory tract infection
57%
Pyrexia
43%
Transaminases increased
43%
Cough
43%
Nausea
43%
Platelet count decreased
29%
Diarrhoea
29%
Abdominal pain
29%
Nasopharyngitis
14%
Viral skin infection
14%
Aspartate aminotransferase increased
14%
Subglottic laryngitis
14%
Liver function test increased
14%
Decreased appetite
14%
Pericardial effusion
14%
Periorbital swelling
14%
Constipation
14%
Urinary tract infection
14%
Alanine aminotransferase increased
14%
Gastroenteritis viral
14%
Stoma site erythema
14%
Blood lactate dehydrogenase increased
14%
COVID-19
14%
Blood urea increased
14%
Torus fracture
14%
Conjunctivitis
14%
Respiratory tract infection
14%
Hypertransaminasaemia
14%
Gastroenteritis
14%
Cushing's syndrome
14%
Thrombocytopenia
14%
Hip deformity
14%
Eczema infantile
14%
Lethargy
14%
Rash
14%
Upper gastrointestinal haemorrhage
14%
Osteopenia
14%
Hallucination
14%
Hypersensitivity
14%
Gastritis
14%
Joint dislocation
14%
Dehydration
14%
Oropharyngeal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
OAV101 1.1e14 vg/kg 8.5 to 13 kg
OAV101 1.1e14 vg/kg Greater Than 17 to 21 kg
OAV101 1.1e14 vg/kg Greater Than 13 to 17 kg
OAV101 1.1e14 vg/kg Overall

STEER Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: OAV101Experimental Treatment1 Intervention
OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10^14 vector genomes (vg).
Group II: Sham controlPlacebo Group1 Intervention
A skin prick in the lumbar region without any medication.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
OAV101
2021
Completed Phase 4
~40

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,855 Previous Clinical Trials
4,197,229 Total Patients Enrolled

Media Library

OAV101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05089656 — Phase 3
Spinal Muscular Atrophy Research Study Groups: OAV101, Sham control
Spinal Muscular Atrophy Clinical Trial 2023: OAV101 Highlights & Side Effects. Trial Name: NCT05089656 — Phase 3
OAV101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05089656 — Phase 3

Frequently Asked Questions

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

What are the odds of OAV101 having negative consequences for patients?

"OAV101's safety is estimated to be a 3. This evaluation comes from the fact that OAV101 is in Phase 3 trials, thus meaning there is both efficacy and safety data available."

Answered by AI

How do I know if I am eligible to participate in this particular research project?

"Eligible participants for this study will have muscular atrophy and be aged between 2 to 17 years old. The researchers hope to enroll 125 patients in total."

Answered by AI

Are there any patients still being accepted into this trial?

"Yes, as can be seen on clinicaltrials.gov, this study is still recruiting patients. The listing was created on February 1st, 2022 and updated just last week on October 28th, 2022."

Answered by AI

Are patients who are over 55 years old still able to sign up for this clinical trial?

"In order to be included in this trial, patients must fall between the age of 2 and 17. Out of the 201 total trials, 49 are for people under 18 and 152 are for senior citizens."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Novartis Investigative Site
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
2022. "Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05089656.
All > Spinal Muscular Atrophy
~26 spots leftby Dec 2024
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