NCT07265232WithdrawnPhase 3
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Source: ClinicalTrials.gov · Last updated 25 Sept 2026
Reason given: Study withdrawn prior to participant enrollment following a reassessment of the clinical development plan.
Brief summary (registry, in English)
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.
- Status (registry)
- Withdrawn
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 0 (actual)
- First submitted
- 21 Nov 2025
- Start date
- 15 Oct 2025
- Primary completion date
- 15 Oct 2030
- Lead sponsor
- Lantu Biopharma
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07265232
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- None
Who the study is looking for
- 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.
Age in the registry: 6 months to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Tzu chi hospital | Jakarta | Indonesia | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.