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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.
Must not have
Requiring invasive ventilation, awake noninvasive ventilation for > 6 hours during a 24-hour period, noninvasive ventilation for > 12 hours during a 24-hour period or requiring tracheostomy
Anti-adeno-associated virus serotype 9 (AAV9) antibody titer reported as elevated (reference to > 1:50 or validated result consistent with being elevated) at screening as determined by sponsor designated lab
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to 64 weeks
Awards & highlights
Pivotal 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.
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.
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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I need a machine to help me breathe, either through a mask for more than 6 hours a day or through a tracheostomy.
Select...
My test shows high levels of AAV9 antibodies.
Select...
I do not have severe joint stiffness or a significant spine curve that would affect movement tests.
Select...
My liver tests are higher than normal.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to 64 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 64 weeks 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 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 study objectives
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 4 trial • 16 Patients • NCT05073133
44%
Pyrexia
44%
Vomiting
31%
Alanine aminotransferase increased
31%
Aspartate aminotransferase increased
31%
Gamma-glutamyltransferase increased
25%
Cough
25%
Platelet count decreased
19%
Bronchiolitis
19%
Influenza
19%
Respiratory tract infection
19%
Hepatic enzyme increased
13%
Bilirubin conjugated increased
13%
Blood lactate dehydrogenase increased
13%
Transaminases increased
13%
Blood alkaline phosphatase increased
13%
Rhinorrhoea
13%
Upper respiratory tract congestion
13%
Gastroenteritis
13%
Irritability
13%
Viral infection
13%
Diarrhoea
13%
Thrombotic microangiopathy
13%
COVID-19
13%
Catarrh
13%
Tonsillitis
6%
Rhinitis
6%
Rash
6%
Coxsackie viral infection
6%
Pneumonia
6%
Constipation
6%
Subcutaneous emphysema
6%
Hepatic failure
6%
Thrombocytopenia
6%
White blood cell count increased
6%
Hypoglycaemia
6%
Upper respiratory tract infection
6%
Nausea
6%
Decreased appetite
6%
Renal failure
6%
Femur fracture
6%
Salivary hypersecretion
6%
Laryngitis
6%
Pneumonia aspiration
6%
Asymptomatic bacteriuria
6%
Hypersensitivity
6%
Pneumonia bacterial
6%
Conjunctivitis
6%
Stomatitis
6%
Urinary tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
OAV101A1 1.le 14vg/kg All

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
2,921 Previous Clinical Trials
4,254,185 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
~33 spots leftby Dec 2025
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