Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Recruiting
Phase 3
Interventional Study
Spinal Muscular Atrophy (SMA)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 Months and older
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 15 (estimated)
- Sponsor
- Lantu Biopharma · Industry
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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 e…
Trial Locations
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Eligibility 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 l…
Contacts
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