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

Lantu Biopharma

Status and phase: Enrolling; Phase 3

Conditions: Spinal Muscular Atrophy (SMA)

Treatments: Biological: vesemnogene lantuparvovec

Study type: Interventional

Funder types: Industry

Identifiers

NCT07265232

HR 25-02

Details and patient eligibility

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.

Full description

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 No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.

Enrollment: 15 estimated patients

Sex: All

Ages: 6+ months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

The selected dose inchildren ≥ 6 months of age

Experimental group

Description:

Administration the selected dose of Vesemnogene Lantuparvovec in children \> 6 months of age

Treatment:

Biological: vesemnogene lantuparvovec