OAV101 for Spinal Muscular Atrophy

(STEER Trial)

Recruiting at 41 trial locations

Overseen By Novartis Medical Information - International

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Must not be taking: SMN-targeting therapies

Disqualifiers: Elevated AAV9 antibodies, Hepatic dysfunction, Ventilation needs, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

127 Participants Needed

Trial Summary

What is the purpose of this trial?

To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to \< 18 years of age over a 15 month trial duration.

Eligibility Criteria

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.

Inclusion Criteria

My condition is confirmed to be 5q spinal muscular atrophy.

I can sit by myself but have never been able to walk on my own.

I have never taken SMN-targeting therapies like Evrysdi or Spinraza.

Exclusion Criteria

I need a machine to help me breathe, either through a mask for more than 6 hours a day or through a tracheostomy.

You have significant problems with feeling or sensation in your nerves during a medical exam.

My test shows high levels of AAV9 antibodies.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

6-8 weeks

Treatment Period 1

Participants receive OAV101 or sham procedure with in-patient hospitalization on Study Day 1, Day 2, and Day 3 (optional)

1 week

3 visits (in-patient)

Follow-up Period 1

Participants are monitored for safety and efficacy assessments

52 weeks

Treatment Period 2

Eligible participants receive OAV101 or sham procedure with in-patient hospitalization

1 week

3 visits (in-patient)

Follow-up Period 2

Participants are monitored for safety and efficacy assessments

12 weeks

Long-term follow-up

Participants may enroll in a long-term follow-up study to monitor long-term safety and efficacy

15 years

Treatment Details

Interventions

• OAV101
• Sham control

Trial Overview 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.

Participant Groups

Group I: OAV101 Experimental Treatment 1 Intervention

OAV101 administered as a single, one-time intrathecal dose of 1.2 x 10\^14 vector genomes (vg).

Group II: Sham control Placebo Group 1 Intervention

A skin prick in the lumbar region without any medication.

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD