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

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.

Study Overview

• Status: Recruiting
• Conditions: Spinal Muscular Atrophy (SMA)
• Intervention / Treatment: Biological: vesemnogene lantuparvovec

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 No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 3

Contacts and Locations

Study Locations

• Indonesia
  • Indonesia
    • Jakarta, Indonesia, Indonesia, 14470
      • Recruiting
      • Tzu chi hospital
      • Contact: Putri; Phone Number: +62 21 50950888; Email: pedonco.tch@tzuchihospital.co.id

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: The selected dose inchildren ≥ 6 months of age; Administration the selected dose of Vesemnogene Lantuparvovec in children > 6 months of age	Biological: vesemnogene lantuparvovec; Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Baseline up to 5 years old

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	Baseline up to 5 vears old

Collaborators and Investigators

• Sponsor: Lantu Biopharma

Study record dates

Study Major Dates

• Study Start (Actual): October 15, 2025
• Primary Completion (Estimated): October 15, 2030
• Study Completion (Estimated): October 15, 2030

Study Registration Dates

• First Submitted: November 21, 2025
• First Submitted That Met QC Criteria: December 2, 2025
• First Posted (Estimated): December 4, 2025

Study Record Updates

• Last Update Posted (Estimated): December 4, 2025
• Last Update Submitted That Met QC Criteria: December 2, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy, Spinal
• Other Study ID Numbers: HR 25-02

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No