Actively Recruiting
Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy
Led by Lantu Biopharma · Updated on 2025-07-22
20
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety and effectiveness of a gene therapy called AAV-hSMN1 (vesemnogene lantuparvovec) in patients with spinal muscular atrophy (SMA), a progressive genetic condition. The study includes children both younger and older than 24 months and is divided into two stages to explore appropriate dosing and treatment effects in these age groups. Participants will receive one of two escalating doses of the gene therapy if they are younger than 24 months, or the selected dose if they are 24 months or older. The treatment is given on a single day during the study, which is conducted in two stages: Stage 1 for dose escalation in younger children and Stage 2 for the selected dose in older children. Each participant will be involved for about 25 months, starting with a 30-day screening period followed by a 24-month observation and follow-up period. Visits occur frequently during the first month after treatment, then at months 2, 3, 6, 12, 18, and 24. Remote virtual visits and local pediatrician visits are allowed for participants from overseas. Researchers will monitor safety by tracking adverse events and evaluate motor development and function using standardized tests.
CONDITIONS
Brief 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 spinal muscular atrophy confirmed by genetic testing showing bi-allelic SMN1 mutations (deletion or point mutations).
- Patients or their parent(s)/legal guardian(s) willing and able to provide informed consent and comply with study visits and procedures.
You will not qualify if you...
- Anti-AAV9 antibody titers greater than 1:20 as measured by ELISA assay.
- 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%.
- Any other illness or medication judged by the investigator to add unnecessary risk for gene therapy.
- Clinically significant abnormal laboratory test results.
- Participation in recent spinal muscular atrophy treatment trials deemed risky 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, presence of implanted shunt or central venous catheter.
- For children 24 months or older: severe contractures limiting functional assessments or dosing.
- For children 24 months or older: severe scoliosis with spine curvature 50 degrees or more.
- For children 24 months or older: previous, planned, or expected scoliosis surgery within one year of dosing.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Approximately 30 days
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 1 day
Participants receive a single dose of vesemnogene lantuparvovec gene therapy.
1 treatment day (in-person)
Duration - Approximately 24 months
Participants are observed with frequent visits to monitor safety and efficacy after treatment.
Twice weekly visits for the first month post dose, then visits at months 2, 3, 6, 12, 18, and 24 post infusion
Duration - Approximately 24 months
Participants have continued follow-up visits for long-term monitoring after the study observation period.
Follow-up visits with participant's own pediatrician and virtual visits as allowed
Trial Site Locations
Total: 1 location
1
Kunming Hope of Health Hospital
Kunming, Yunnan, China, 650200
Actively Recruiting
Research Team
A
Austin Gao, PhD
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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