Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

ClinicalTrials.gov Identifier: NCT04851873

Novartis Reference Number: COAV101A12306

Last Update: May 20, 2022

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.

Condition 
Spinal Muscular Atrophy
Phase 
Phase 3
Overall status 
Recruiting
Start date 
Sep 08, 2021
Completion date 
Aug 01, 2023
Gender 
All
Age(s)
17 Years and older (Child)

Interventions

Genetic
OAV101
Gene Therapy

Eligibility Criteria

Inclusion

Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.
Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2
Naive to treatment or have discontinued an approved drug/therapy

Exclusion:

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
Anti-Adeno-associated virus serotype 9 (AAV9) antibody titer > 1:50 as determined by ligand binding immunoassay at the time of screening
History of gene therapy, hematopoietic transplantation, or solid organ transplantation

Study Locations

United States
Novartis Investigative Site
Recruiting
Boston, 02115
Massachusetts
United States
Novartis Investigative Site
Recruiting
Saint Louis, 63110
Missouri
United States
Australia
Novartis Investigative Site
Recruiting
Randwick, 2031
New South Wales
Australia
Novartis Investigative Site
Recruiting
Parkville, 3052
Victoria
Australia
Belgium
Novartis Investigative Site
Recruiting
Leuven, 3000
-
Belgium
France
Novartis Investigative Site
Recruiting
Garches, 92380
-
France
Novartis Investigative Site
Recruiting
Strasbourg Cedex, F 67098
-
France
Taiwan
Novartis Investigative Site
Recruiting
Kaohsiung, 80756
-
Taiwan
Novartis Investigative Site
Recruiting
Taipei, 10002
-
Taiwan
United Kingdom
Novartis Investigative Site
Recruiting
London, WC1N 3JH
-
United Kingdom
Novartis Investigative Site
Recruiting
Newcastle Upon Tyne, NE1 4LP
-
United Kingdom

Contacts

Name: 
Novartis Gene Therapies Medical Information (US, Asia-Pacific, Latin America, and Canada)
Name: 
Novartis Gene Therapies EMEA Medical Information (Europe, Middle East and Africa)

Have a question?

Call 1-888-669-6682 or email [email protected]