Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: vesemnogene lantuparvovec.
- Who it may be relevant to
- Registry conditions: Spinal Muscular Atrophy (SMA). Basic parameters: from 6 months · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Indonesia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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 No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.
Interventions
- Biological vesemnogene lantuparvovec
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
Primary outcome measures
- Numbers of participants with adverse events (AEs), serious adverse events (SAEs) [Time frame: Baseline up to 5 years old]
Secondary outcome measures (2)
- Change from baseline in developmental gross motor milestones achieved according to WHO criteria [Time frame: Baseline up to 5 years old]
- Event-free Survival until the 5th year or the last follow-up [Time frame: Baseline up to 5 vears old]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Indonesia · 1 center
- Tzu chi hospital — Jakarta
Identifiers
NCT: NCT07265232 · HR 25-02