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Tests treatment safety and results for Spinal Muscular Atrophy (SMA)
Official title Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
ClinicalTrials.gov ID: NCT07265232
What this study is testing
What is vesemnogene lantuparvovec?
vesemnogene lantuparvovec is an investigational medicine, being studied as a potential treatment for spinal muscular atrophy (sma).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- 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.
You likely can't join if
- None
See the full eligibility 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.
- None
The study team makes the final eligibility decision.
Where it's taking place
- Jakarta, Indonesia, Indonesia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Jakarta, Indonesia, Indonesia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.