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

NCT07265232 | Recruiting Phase 3 DMC

• 1 other identifier: HR 25-02
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Spinal Muscular Atrophy (SMA)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

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

  Baseline up to 5 years old

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

  Baseline up to 5 vears old

Study Arms (1)

The selected dose inchildren ≥ 6 months of age

EXPERIMENTAL

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

Biological: vesemnogene lantuparvovec

Interventions

vesemnogene lantuparvovec BIOLOGICAL

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

The selected dose inchildren ≥ 6 months of age

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Lantu Biopharma: lead

Study Sites (1)

Tzu chi hospital

Jakarta, Indonesia, 14470, Indonesia

RECRUITING

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 21, 2025
• First Posted: December 4, 2025
• Study Start: October 15, 2025
• Primary Completion (Estimated): October 15, 2030
• Study Completion (Estimated): October 15, 2030
• Last Updated: December 4, 2025

Record last verified: 2025-12

Locations

ID (1)