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

NCT ID: NCT07265232

Last Updated: 2025-12-04

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-10-15
• Study Completion Date: 2030-10-15

Brief Summary

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.

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 No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.

Conditions

Spinal Muscular Atrophy (SMA)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

The selected dose inchildren ≥ 6 months of age

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

Group Type: EXPERIMENTAL

vesemnogene lantuparvovec

Intervention Type: BIOLOGICAL

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

Interventions

vesemnogene lantuparvovec

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Lantu Biopharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Tzu chi hospital

Jakarta, Indonesia, Indonesia

Site Status: RECRUITING

Countries

Indonesia

Facility Contacts

Putri

Role: primary

pedonco.tch@tzuchihospital.co.id

+62 21 50950888

Other Identifiers

HR 25-02

Identifier Type: -

Identifier Source: org_study_id