EUCTR2020-005995-37-BE
Active, not recruiting
Phase 1
A Phase lllb, 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) - SMART
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- ovartis Pharma AG
- Enrollment
- 24
- Status
- Active, not recruiting
- Last Updated
- 3 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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
- •other protocol\-defined inclusion criteria may apply
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 24
- •F.1\.2 Adults (18\-64 years) no
- •F.1\.2\.1 Number of subjects for this age range
- •F.1\.3 Elderly (\>\=65 years) no
- •F.1\.3\.1 Number of subjects for this age range
Exclusion Criteria
- •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
- •other protocol\-defined exclusion criteria may apply
Outcomes
Primary Outcomes
Not specified
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