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

NCT07265232 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-12-04

No results posted yet for this study

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)

Interventions

BIOLOGICAL

vesemnogene lantuparvovec

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

Sponsors & Collaborators

  • Lantu Biopharma

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-15
Primary Completion
2030-10-15
Completion
2030-10-15

Countries

  • Indonesia

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07265232 on ClinicalTrials.gov