Spinal Muscular Atrophy (SMA)
Conditions
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.
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 No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.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
BIOLOGICALvesemnogene lantuparvovec
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
Sponsors
Lantu Biopharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Numbers of participants with adverse events (AEs), serious adverse events (SAEs) | Baseline up to 5 years old | Participants are monitored for safety from baseline up to the end of the follow-up period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in developmental gross motor milestones achieved according to WHO criteria | Baseline up to 5 years old | For patients with SMA, the percentage of participants who are able to preserve ambulatory function. |
| Event-free Survival until the 5th year or the last follow-up | 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. |
Countries
Indonesia
Outcome results
None listed