Real-world use of vesemnogene lantuparvovec for spinal muscular atrophy in low- and middle-income countries
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
This project sees if vesemnogene lantuparvovec is safe and helps improve motor skills and survival for people with genetically confirmed SMA who cannot access other curative treatments.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 6 Months and up |
| Sex | All |
| Sponsor | Lantu Biopharma Industry-sponsored |
| Locations | 1 site (Jakarta, Indonesia) |
| Trial ID | NCT07265232 on ClinicalTrials.gov |
What this trial studies
This is an observational real-world registry following patients with genetically confirmed spinal muscular atrophy who receive vesemnogene lantuparvovec at a participating center. Eligible patients are screened for anti-AAV antibody levels, adequate liver function, and suitability for intrathecal administration before treatment. After administration, patients are monitored over time for developmental gross motor milestones, survival, laboratory values, and adverse events including predefined events of special interest. No subjects are actively withdrawn by the protocol, and follow-up reflects routine clinical visits and reporting.
Who should consider this trial
Good fit: Ideal candidates are people with genetically confirmed SMA who have low anti-AAV antibody titers (<1:20), acceptable liver tests, no contraindication to intrathecal procedures, and who are unable to access or have failed other curative SMA treatments.
Not a fit: Patients with high anti-AAV antibody levels, significant liver dysfunction, contraindications to intrathecal delivery, or those already doing well on available curative therapies are unlikely to benefit from this treatment pathway.
Why it matters
Potential benefit: If successful, the program could show that vesemnogene lantuparvovec improves motor development and survival and has an acceptable safety profile in real-world LMIC settings, supporting broader access.
How similar studies have performed: Related AAV-based SMN gene therapies have produced substantial improvements in motor milestones and survival in clinical trials and real-world use, but data specifically for vesemnogene lantuparvovec in LMIC populations are limited.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Jakarta, Indonesia
- Tzu chi hospital — Jakarta, Indonesia, Indonesia (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.