Spinal Muscular Atrophy (SMA)Phase 3NCT07265232
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
A Phase 3 study testing vesemnogene lantuparvovec in people with Spinal Muscular Atrophy (SMA), run by Lantu Biopharma at 1 site in Indonesia.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Spinal Muscular Atrophy (SMA)
- What is being tested?
- vesemnogene lantuparvovec
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteIndonesia
- Study dates
- 15 Oct 2025to 15 Oct 2030
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willing and able to give written informed consent for participation in the study.
- Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
- SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
- Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
- Total AAV antibody titres < 1:20 as determined by ELISA assay.
- Normal liver function (AST/ALT < 3XULN, Bilirubin <3.0 mg/dL).
- Unable to access or failure to respond to currently available curative treatments for SMA.
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
About this study
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 events reporting and laboratory tests, and monitoring of Adverse events of special interest.
Read the detailed description
This is an observational study designed to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for SMA. Potential patients with genetic diagnosis of SMA will be evaluated for eligibility to undergo available gene therapies. Following the administration of Vesemnogene therapy, patient will be monitored for toxicity and response to treatment. No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.
- Conditions
- Spinal Muscular Atrophy (SMA)
- 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
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.
| Study group | Role | What they receive |
|---|---|---|
| The selected dose inchildren ≥ 6 months of age | Experimental | Administration the selected dose of Vesemnogene Lantuparvovec in children > 6 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 5 years old
Secondary outcomes (2)
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 5 years old
Event-free Survival until the 5th year or the last follow-up
Event-free survival was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study until the 5th year or the last follow-up.
Measured: Baseline up to 5 vears old
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2025
- Study start15 Oct 2025 (estimated)
- Primary completion15 Oct 2030 (estimated)
- Study completion15 Oct 2030 (estimated)
Why it stopped: Study withdrawn prior to participant enrollment following a reassessment of the clinical development plan.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy (SMA)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lantu Biopharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07265232
- Sponsor's protocol ID
- HR 25-02
- Status last verified
- Sep 2026
- First submitted
- 21 Nov 2025
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
- ClinicalTrials.gov ID
- NCT07265232
- Acronym / sponsor ID
- HR 25-02
- Conditions
- Spinal Muscular Atrophy (SMA)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study withdrawn prior to participant enrollment following a reassessment of the clinical development plan.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Lantu BiopharmaIndustry (pharma, biotech, devices)
- Study start
- 15 Oct 2025 (estimated)
- Primary completion
- 15 Oct 2030 (estimated)
- Study completion
- 15 Oct 2030 (estimated)
- First posted
- 4 Dec 2025 (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.
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 events reporting and laboratory tests, and monitoring of Adverse events of special interest.
Detailed description
This is an observational study designed to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for SMA. Potential patients with genetic diagnosis of SMA will be evaluated for eligibility to undergo available gene therapies. Following the administration of Vesemnogene therapy, patient will be monitored for toxicity and response to treatment. No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| The selected dose inchildren ≥ 6 months of age | Experimental | Biological: vesemnogene lantuparvovecAdministration the selected dose of Vesemnogene Lantuparvovec in children > 6 months of age |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Willing and able to give written informed consent for participation in the study.
- Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
- SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
- Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
- Total AAV antibody titres < 1:20 as determined by ELISA assay.
Key exclusion criteria (1 listed)
- None
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 lantuparvovecExploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA. | Biological | The selected dose inchildren ≥ 6 months of age |
Primary outcome
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
Time frame: Baseline up to 5 years old
Participants are monitored for safety from baseline up to the end of the follow-up period.
2 secondary outcomes
Change from baseline in developmental gross motor milestones achieved according to WHO criteria
Time frame: Baseline up to 5 years old
Event-free Survival until the 5th year or the last follow-up
Time frame: Baseline up to 5 vears old
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 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 3 study testing vesemnogene lantuparvovec in people with Spinal Muscular Atrophy (SMA), run by Lantu Biopharma at 1 site in Indonesia.
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 7 inclusion and 1 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: withdrawn, as of the sponsor's last update on 25 Sep 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 Indonesia. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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 5 years old). 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 25 Sep 2026.