Actively Recruiting
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Led by Lantu Biopharma · Updated on 2025-12-04
15
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 less than 1:20 as determined by ELISA assay.
- Normal liver function (AST/ALT less than 3 times upper limit of normal, Bilirubin less than 3.0 mg/dL).
- Unable to access or failure to respond to currently available curative treatments for SMA.
You will not qualify if you...
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AI-Screening
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Trial Site Locations
Total: 1 location
1
Tzu chi hospital
Jakarta, Indonesia, Indonesia, 14470
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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