Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

Recruiting now · Phase 3

Conditions studied: Spinal Muscular Atrophy (SMA)

In brief

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.

Key facts

Study ID

NCT07265232

Run by

Lantu Biopharma

People needed

15

Starts

2025-10-15

Expected to finish

2030-10-15

Last updated by the study team

2025-12-04

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• None

Where it is running

• Tzu chi hospital — Jakarta, Indonesia, Indonesia (enrolling)

Full record on ClinicalTrials.gov

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