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Can Gene-Modified stem cells stop a devastating brain disease?

NCT ID NCT07046338

Cure Sponsor: Shenzhen Geno-Immune Medical Institute Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 01, 2026 · Last updated Sep 02, 2026 · Updated 1 time

Summary

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare inherited disease that damages the nervous system. Researchers take a patient's own blood stem cells, add a corrected gene, and infuse them back. The goal is to see if this approach is safe and can slow or stop disease progression. The study involves 10 participants, including infants and children.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Lentiviral TYF-ARSA modified autologous hematopoietic stem cells
What this could lead to
If successful, this could provide a one-time treatment that corrects the genetic cause of MLD, potentially halting or slowing the disease.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions or the modified cells not engrafting.

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

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 10 people

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

Expected to start

Jun 2027

An estimate. Start dates often move.

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 month to 50 years

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. age \>= 1 month 2. ARSA gene sequence analysis to confirm MLD mutations 3. Brain MR Imaging 4. Parent / guardian / patient signing informed consent 5. Patients and their families have a strong willingness to participate in clinical trials, are willing to bear all the consequences caused by the failure of the trial, and sign the informed consent Exclusion Criteria: 1. HIV positive 2. Experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency 3. Cannot perform an MRI 4. Infection or dermatosis at infusion site 5. Any condition that may increase the subject's risk or interfere with the results of the trial, e.g. in addition to MLD, there are other neurological disorders.

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Conditions

The condition(s) this trial relates to.

metachromatic leukodystrophy

As listed by the trial registrant

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

Metachromatic leukodystrophy (MLD)

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

  • Shenzhen Geno-Immune Medical Institute

    Shenzhen, Guangdong, 518000, China

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