RECRUITINGPhase 3INTERVENTIONAL
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
1. Willing and able to give written willing to sign a consent form 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.
Who Should NOT Join This Trial:
None
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
BIOLOGICAL
vesemnogene lantuparvovec
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
Locations (1)
Tzu chi hospital
Jakarta, Indonesia, Indonesia