Spinal Muscular AtrophyPhase 1/2NCT06288230
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
A Phase 1/2 study testing vesemnogene lantuparvovec in people with Spinal Muscular Atrophy, run by Lantu Biopharma at 1 site in China.
Trial summary
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- vesemnogene lantuparvovec
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 20 Oct 2024to 30 Oct 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
Read the detailed description
Study duration per participant is approximately 25 months including an approximately 30-day screening/baseline period, an approximately 24-month study observation period including 1 treatment day, and an approximately 24-month follow-up period. Patients will be stratified in two groups, those < 24 months of age at time of dosing and those ≥ 24 months of age at time of dosing. This study will be conducted in 2 stages:
Stage 1: dose escalting study in children <24 months of age. Stage 2: the selecetd dose from Stage 1 in children ≥ 24 months of age.
Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose, and followed by visits at months 2, 3, 6 12, 18 and 24 post infusion. Unscheduled visits may occur if the investigator determines that they are necessary.
For patients enrolled from overseas, follow-up visits with patient's own paediatrician together with remote virtual visits are allowed.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- vesemnogene lantuparvovec
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vesemnogene lantuparvovecBiological
Also called AAV-hSMN1
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
| Study group | Role | What they receive |
|---|---|---|
| Stage 1: Dose A in children < 24 months of age | Experimental | Dose escalting study: Administration Dose A of Vesemnogene Lantuparvovec in children < 24 months of age Receives: Biological: vesemnogene lantuparvovec |
| Stage 1: Dose B in children < 24 months of age | Experimental | Dose escalting study: Administration Dose B of Vesemnogene Lantuparvovec in children < 24 months of age Receives: Biological: vesemnogene lantuparvovec |
| Stage 2: The selected dose in children ≥ 24 months of age | Experimental | Administration the selected dose of Vesemnogene Lantuparvovec in children ≥ 24 months of age Receives: Biological: vesemnogene lantuparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
Participants are monitored for safety from baseline up to the end of the follow-up period.
Measured: Baseline up to 24 months
Secondary outcomes (3)
Change from baseline in developmental gross motor milestones achieved according to WHO criteria
For patients with SMA, the percentage of participants who are able to preserve ambulatory function.
Measured: Baseline up to 24 months
Change from baseline in CHOP-INTEND in children <24 months of age
The CHOP-INTEND contains 16 items rated from 0 to 4. Total scores range from 0-64. Higher scores indicate higher levels of motor ability.
Measured: Baseline up to 24 months
Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) in children ≥ 24 months of age
The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
Measured: Baseline up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2024
- Study start20 Oct 2024
- Primary completion30 Oct 2027 (estimated)
- Study completion30 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lantu Biopharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06288230
- Sponsor's protocol ID
- LT01-101
- Status last verified
- Jun 2026
- First submitted
- 18 Feb 2024
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT06288230
- Acronym / sponsor ID
- LT01-101
- Conditions
- Spinal Muscular Atrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Lantu BiopharmaIndustry (pharma, biotech, devices)
- Study start
- 20 Oct 2024 (actual)
- Primary completion
- 30 Oct 2027 (estimated)
- Study completion
- 30 Oct 2027 (estimated)
- First posted
- 1 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
Detailed description
Study duration per participant is approximately 25 months including an approximately 30-day screening/baseline period, an approximately 24-month study observation period including 1 treatment day, and an approximately 24-month follow-up period. Patients will be stratified in two groups, those < 24 months of age at time of dosing and those ≥ 24 months of age at time of dosing. This study will be conducted in 2 stages:
Stage 1: dose escalting study in children <24 months of age. Stage 2: the selecetd dose from Stage 1 in children ≥ 24 months of age.
Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose, and followed by visits at months 2, 3, 6 12, 18 and 24 post infusion. Unscheduled visits may occur if the investigator determines that they are necessary.
For patients enrolled from overseas, follow-up visits with patient's own paediatrician together with remote virtual visits are allowed.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Stage 1: Dose A in children < 24 months of age | Experimental | Biological: vesemnogene lantuparvovecDose escalting study: Administration Dose A of Vesemnogene Lantuparvovec in children < 24 months of age |
| Stage 1: Dose B in children < 24 months of age | Experimental | Biological: vesemnogene lantuparvovecDose escalting study: Administration Dose B of Vesemnogene Lantuparvovec in children < 24 months of age |
| Stage 2: The selected dose in children ≥ 24 months of age | Experimental | Biological: vesemnogene lantuparvovecAdministration the selected dose of Vesemnogene Lantuparvovec in children ≥ 24 months of age |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vesemnogene lantuparvovecAlso: AAV-hSMN1Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA. | Biological | Stage 1: Dose A in children < 24 months of age, Stage 1: Dose B in children < 24 months of age, Stage 2: The selected dose in children ≥ 24 months of age |
Primary outcome
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
Time frame: Baseline up to 24 months
Participants are monitored for safety from baseline up to the end of the follow-up period.
3 secondary outcomes
Change from baseline in developmental gross motor milestones achieved according to WHO criteria
Time frame: Baseline up to 24 months
Change from baseline in CHOP-INTEND in children <24 months of age
Time frame: Baseline up to 24 months
Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) in children ≥ 24 months of age
Time frame: Baseline up to 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing vesemnogene lantuparvovec in people with Spinal Muscular Atrophy, run by Lantu Biopharma at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Numbers of participants with adverse events (AEs), serious adverse events (SAEs) (time frame: Baseline up to 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2026.