Actively Recruiting
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
Led by Lantu Biopharma · Updated on 2025-07-22
20
Participants Needed
1
Research Sites
157 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
CONDITIONS
Official Title
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of SMA confirmed by genetic testing showing bi-allelic SMN1 mutations (deletion or point mutations)
- Patient or parent/legal guardian willing and able to provide informed consent and follow study procedures and visit schedule
You will not qualify if you...
- Anti-AAV9 antibody levels greater than 1:20 by ELISA test
- Active viral infections including HIV or positive hepatitis B or C serology
- Use of invasive ventilatory support such as tracheotomy with positive pressure or oxygen saturation below 95%
- Other illnesses or medications that pose extra risk for gene therapy as judged by the investigator
- Significant abnormal lab test results
- Recent participation in other SMA treatment trials that increase risk as judged by the principal investigator
- Signs of aspiration on swallowing test and refusal to use alternative feeding methods
- For children 24 months or older: contraindications to spinal tap or intrathecal therapy, implanted CSF shunt or central venous catheter
- For children 24 months or older: severe contractures interfering with functional measures or dosing
- For children 24 months or older: severe scoliosis with spine curvature of 50 degrees or more
- For children 24 months or older: previous, planned, or expected scoliosis surgery within 1 year of dosing
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Kunming Hope of Health Hospital
Kunming, Yunnan, China, 650200
Actively Recruiting
Research Team
A
Austin Gao, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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