Gene therapy trial aims to halt Muscle-Wasting disease
NCT ID NCT06288230
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check safety, but researchers will also see if it improves motor skills like sitting or walking.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- vesemnogene lantuparvovec (gene therapy)
- What this could lead to
- If successful, this could provide a new treatment option that improves motor function and slows disease progression in people with spinal muscular atrophy.
- What could go wrong
- This is a small, early-phase trial (Phase 1/2) focused on safety, so it is too soon to know if the therapy works. There may be risks from the gene therapy or the viral delivery system.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
About 20 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Oct 2024
- Expected to finish
-
Oct 2027
An estimate. End dates often move.
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Diagnosis of SMA based on gene mutation analysis with bi-allelic survival motor neuron (SMN1) mutations (deletion or point mutations). * Patients or Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule. Exclusion Criteria: * Anti-AAV9 antibody titers \>1:20 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay. * Active viral infection (includes HIV or serology positive for hepatitis B or C). * Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \<95% saturation. * Concomitant illness and any drug that in the opinion of the investigator creates unnecessary risks for gene transfer. * Clinically significant abnormal laboratory values. * Participation in a recent SMA treatment clinical trial that in the opinion of the PI creates unnecessary risks for gene transfer. * Patient with signs of aspiration based on a swallowing test and unwilling to use an alternative method to oral feeding. * For children ≥ 24 months of age, contraindications for spinal tap procedure or administration of intrathecal therapy or presence of an implanted shunt for the drainage of CSF or an implanted central venous (CNS) catheter. * For children ≥ 24 months of age, severe contractures as determined by Physical Therapist(s) at screening that interfere with either the ability to attain/demonstrate functional measures or interferes with ability to receive dosing. * For children ≥ 24 months of age, severe scoliosis (defined as ≥ 50° curvature of spine) evident on X-ray examination. * For children ≥ 24 months of age, previous, planned or expected scoliosis repair surgery/procedure within 1 year of dose administration.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Kunming Hope of Health Hospital
Kunming, Yunnan, 650200, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can more Hands-On therapy and home devices help children with SMA?
- Can a brain implant let paralyzed people control computers with their minds?
- Can treating spinal muscular atrophy in the womb be safe and helpful? a new registry aims to find out
- A Baby's fidgets may reveal brain health: study tests early warning signs
- Can a muscle-boosting antibody help people with spinal muscular atrophy over the long haul?
- Can a patient registry unlock the secrets of spinal muscular atrophy?