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Gene therapy for SMA tested in real-world settings of poorer nations

NCT ID NCT07265232

Disease control Sponsor: Lantu Biopharma Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is testing a gene therapy called vesemnogene lantuparvovec for spinal muscular atrophy (SMA) in low- and middle-income countries. Researchers will track safety and whether children reach motor milestones like sitting or walking. The trial involves 15 participants and aims to see if the treatment works outside of wealthy countries.

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 (a gene therapy)
What this could lead to
If successful, this could show that gene therapy for SMA is safe and effective in low-resource settings, potentially expanding access to treatment.
What could go wrong
This is a small, early real-world study with only 15 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions and unknown long-term effects.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Oct 2030

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

6 months and older

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Willing and able to give written informed consent for participation in the study. 2. Genetic confirmation of SMA (biallelic deletion or mutation of SMN1). 3. SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial. 4. Absence of contraindications for spinal tap procedure or administration of intrathecal therapy. 5. Total AAV antibody titres \< 1:20 as determined by ELISA assay. 6. Normal liver function (AST/ALT \< 3XULN, Bilirubin \<3.0 mg/dL). 7. Unable to access or failure to respond to currently available curative treatments for SMA. Exclusion Criteria: None

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Spinal muscular atrophy (SMA)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Tzu chi hospital

    RECRUITING

    Jakarta, Indonesia, 14470, Indonesia

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