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Gene Therapy

OAV101 for Spinal Muscular Atrophy (SMART 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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights

SMART Trial Summary

This trial is testing a new drug for safety, tolerability, and efficacy in children with spinal muscular atrophy.

Eligible Conditions
  • Spinal Muscular Atrophy

SMART Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months 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 Vital Signs Measurements - Diastolic Blood Pressure (mmHg)
Change From Baseline in Vital Signs Measurements - Oxygen Saturation Level
Change From Baseline in Vital Signs Measurements - Pulse Rate (Beats/Min)
+7 more
Secondary outcome measures
Achievement of Development Motor Milestones According to the Modified and Combined WHO-MGRS and Bayley Scale of Infant and Toddler Development.
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as Appropriate According to Participant Age
Change From Baseline in Revised Upper Limb Module (RULM), as Appropriate According to Participant Age.

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%
Conjunctivitis
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%
Torus fracture
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%
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

SMART Trial Design

1Treatment groups
Experimental Treatment
Group I: OAV101Experimental Treatment1 Intervention
Participants received a single IV dose administration of OAV101
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
2,857 Previous Clinical Trials
4,197,777 Total Patients Enrolled

Frequently Asked Questions

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

How does OAV101 increase the risk for patients?

"OAV101 received a safety score of 3 because it is a Phase 3 trial. This means that, while there is still some data yet to be collected, there is evidence supporting its efficacy and multiple rounds of data affirming its safety."

Answered by AI

Are researchers recruiting more participants for this trial?

"The clinicaltrials.gov website indicates that this study is still looking for participants. The listing was first created on September 8th, 2021 and was last updated on October 2nd, 2022."

Answered by AI

Does this scientific research include elderly participants aged 85 or above?

"The age bracket that this study is recruiting from is 3 months old to 17 years old."

Answered by AI

Who would be an ideal candidate to join this clinical trial?

"This clinical trial is recruiting 24 participants with atrophy aged 3 Months and 17. Most notable, patients are required to meet the following criteria: Naive to treatment or have discontinued an approved drug/therapy, Previous OAV101 use or previous use of any AAV9 gene therapy, Body Mass Index (BMI) < 3rd percentile based on World Health Organization (WHO) Child Growth Standard, Participant with history of aspiration pneumonia or signs of aspiration (eg, coughing or sputtering of food) within 4 weeks prior to screening, History of gene therapy, hematopoietic transplantation, or solid organ transplant"

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
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
2021. "Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04851873.
~7 spots leftby Apr 2025
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