Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Lantu Biopharma
15 participants
Oct 15, 2025
INTERVENTIONAL
Conditions
Summary
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival. * To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.
Eligibility
Inclusion Criteria7
- Willing and able to give written informed consent for participation in the study.
- Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
- SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
- Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
- Total AAV antibody titres \< 1:20 as determined by ELISA assay.
- Normal liver function (AST/ALT \< 3XULN, Bilirubin \<3.0 mg/dL).
- Unable to access or failure to respond to currently available curative treatments for SMA.
Exclusion Criteria1
- None
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Interventions
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
Locations(1)
View Full Details on ClinicalTrials.gov
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NCT07265232