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

Lantu Biopharma

Overview

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.

This is an observational study designed to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for SMA. Potential patients with genetic diagnosis of SMA will be evaluated for eligibility to undergo available gene therapies. Following the administration of Vesemnogene therapy, patient will be monitored for toxicity and response to treatment. No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Spinal Muscular Atrophy (SMA)

Interventions

vesemnogene lantuparvovecBIOLOGICAL

Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.

Eligibility

Sex: ALLMin age: 6 Months

Medical Language ↔ Plain English

Inclusion Criteria: 1. Willing and able to give written informed consent for participation in the study. 2. Genetic confirmation of SMA (biallelic deletion or mutation of SMN1). 3. SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial. 4. Absence of contraindications for spinal tap procedure or administration of intrathecal therapy. 5. Total AAV antibody titres \< 1:20 as determined by ELISA assay. 6. Normal liver function (AST/ALT \< 3XULN, Bilirubin \<3.0 mg/dL). 7. Unable to access or failure to respond to currently available curative treatments for SMA. Exclusion Criteria: None

Locations (1)

Tzu chi hospital

Jakarta, Indonesia, Indonesia

Outcomes

Primary Outcomes

Numbers of participants with adverse events (AEs), serious adverse events (SAEs)

Participants are monitored for safety from baseline up to the end of the follow-up period.

Time frame: Baseline up to 5 years old

Secondary Outcomes

Change from baseline in developmental gross motor milestones achieved according to WHO criteria

For patients with SMA, the percentage of participants who are able to preserve ambulatory function.

Time frame: Baseline up to 5 years old

Event-free Survival until the 5th year or the last follow-up

Event-free survival was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study until the 5th year or the last follow-up.

Time frame: Baseline up to 5 vears old

Data from ClinicalTrials.gov

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