Status:
COMPLETED
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE3
Brief Summary
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 neur...
Detailed Description
This was an open-label, single arm, multi-center study designed to evaluate the safety, tolerability and efficacy of OAV101 in participants with SMA who weigh ≥ 8.5 kg and ≤ 21 kg. The study aimed to ...
Eligibility Criteria
Inclusion
- 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 adeno-associated virus serotype 9 (AAV9) gene therapy
- BMI \< 3rd percentile
- Participant with history of aspiration pneumonia or signs of aspiration
- Elevated anti-AAV9 antibody
- History of gene therapy, hematopoietic transplantation, or solid organ transplantation
- Inability to take corticosteroids
- Concomitant use of immunosuppressive therapy
- Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101
- Awake hypoxemia or awake oxygen saturation level decrease
- Hepatic dysfunction
- Presence of a confirmed or suspected infection
- If previously treated with disease modifying therapy, specified washout times apply
- Documented any parental consanguinity.
Exclusion
Key Trial Info
Start Date :
September 8 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 13 2023
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT04851873
Start Date
September 8 2021
End Date
June 13 2023
Last Update
October 9 2024
Active Locations (13)
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1
Novartis Investigative Site
Boston, Massachusetts, United States, 02115
2
Novartis Investigative Site
St Louis, Missouri, United States, 63110
3
Novartis Investigative Site
Randwick, New South Wales, Australia, 2031
4
Novartis Investigative Site
Leuven, Belgium, 3000