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Gene Therapy

OAV101 for SMA (STRENGTH Trial)

Phase 3

Recruiting

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

SMA diagnosis

Aged 2 to 12 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

STRENGTH Trial Summary

This trialstudies the safety of a new medication in children 2-12 years old over one year.

Who is the study for?

This trial is for children aged 2 to 12 with Spinal Muscular Atrophy (SMA) who have previously been treated with nusinersen or risdiplam but stopped. They must show SMA symptoms and not have high antibody levels against AAV9, significant test abnormalities, contraindications for spinal taps, recent vaccinations, hospitalizations for pulmonary events or surgeries planned.Check my eligibility

What is being tested?

The study tests OAV101's safety and effectiveness in treating SMA when given through a spinal injection. It involves one treatment day followed by a year of follow-up. Participants are divided into two age groups: 2-5 years old and 6-12 years old.See study design

What are the potential side effects?

Potential side effects aren't specified here but may include reactions related to the immune system due to antibodies against AAV9 or complications from the lumbar puncture procedure.

STRENGTH Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with Spinal Muscular Atrophy (SMA).

Select...

I am between 2 and 12 years old.

Select...

I have received at least four doses of Spinraza or been on Evrysdi for 3 months.

STRENGTH Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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

Number and percentage of participants reporting AEs, related AEs, SAEs, and AESIs

Secondary outcome measures

Change from baseline to Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score

Change from baseline to Week 52 visit in the HFMSE total score

Change from baseline to Week 52 visit in the RULM total Score

Side effects data

From 2023 Phase 3 trial • 24 Patients • NCT04851873

71%

Vomiting

57%

Pyrexia

57%

Upper respiratory tract infection

43%

Transaminases increased

43%

Nausea

43%

Platelet count decreased

43%

Cough

29%

Diarrhoea

29%

Abdominal pain

29%

Nasopharyngitis

14%

Viral skin infection

14%

Aspartate aminotransferase increased

14%

Conjunctivitis

14%

Torus fracture

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%

Hypertransaminasaemia

14%

Respiratory tract infection

14%

COVID-19

14%

Blood urea increased

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%

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

STRENGTH Trial Design

1Treatment groups

Experimental Treatment

Group I: OAV-101Experimental Treatment1 Intervention

Single intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

OAV101

2021

Completed Phase 4

~40

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor

2,859 Previous Clinical Trials

4,198,216 Total Patients Enrolled

Media Library

OAV101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05386680 — Phase 3

Spinal Muscular Atrophy Research Study Groups: OAV-101

Spinal Muscular Atrophy Clinical Trial 2023: OAV101 Highlights & Side Effects. Trial Name: NCT05386680 — Phase 3

OAV101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05386680 — Phase 3

Frequently Asked Questions

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

Are adults above the age of 18 acceptable candidates for this experiment?

"Taking into account the prerequisites of this clinical trial, juvenile subjects aged between 2 and 12 can enrol."

Answered by AI

Could you kindly explain the risks associated with OAV-101?

"Due to the existence of efficacy data and multiple evidence-based demonstrations confirming its safety, OAV-101 achieved a rating of 3 on our internal scale."

Answered by AI

Is it possible for me to volunteer in this research program?

"To qualify for this trial, applicants must have a diagnosis of spinal muscular atrophy and be between 2-12 years old. Roughly 28 participants are needed overall."

Answered by AI

Is this trial currently open to recruitment?

"Per the information provided on clinicaltrials.gov, this research initiative is actively recruiting patients. The investigation was first initiated on December 1st 2023, and has recently been revised as of January 18th 2023."

Answered by AI

What is the upper limit on recruitment for this research project?

"Yes, according to clinicaltrials.gov, this trial is actively accepting enrolment and was first posted on December 1st 2023. The most recent update took place on January 18th 2023, with the requirement of 28 individuals at a single site."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

2023. "Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05386680.

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