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NCT Number: NCT07265232

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.

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Key information

Conditions

Spinal Muscular Atrophy (SMA) Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

6 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

About this study

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 No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

None

Treatment and study plan

vesemnogene lantuparvovec

Biological

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

Primary outcomes

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

    Time frame: Baseline up to 5 years old

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

Secondary outcomes

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

    Time frame: Baseline up to 5 years old

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

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

    Time frame: Baseline up to 5 vears old

    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.

Sponsors and collaborators

Lead sponsor

Lantu Biopharma

Industry

Registry information

Important dates

Study start
2025
Primary completion
2030
Study completion
2030
First posted
Dec 4, 2025
Registry last updated
Dec 4, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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