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Can a Patient's own stem cells reset the immune system to halt brain damage?

NCT ID NCT07757685

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 11, 2026 · Last updated Aug 12, 2026 · Updated 1 time

Summary

This trial is testing whether a transplant of a patient's own blood-forming stem cells can ease neurological damage caused by hereditary homocysteine remethylation disorders, a rare genetic condition that affects metabolism. The approach aims to reset the immune system and reduce inflammation in the brain. Researchers will monitor safety, how well the transplant works, and changes in neurological function and brain scans. The study includes adults aged 18 to 55 with confirmed diagnosis and neurological symptoms who have not responded well to standard metabolic therapy.

What this could mean

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

Active substance
Autologous hematopoietic stem cell transplantation (using the patient's own stem cells)
What this could lead to
If successful, this approach could offer a way to slow or reverse neurological damage in people with hereditary homocysteine remethylation disorders, potentially improving their quality of life.
What could go wrong
This is an early exploratory study, so safety and effectiveness are not yet proven. The procedure carries risks like infection, organ damage, and failure to collect enough stem cells.

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 50 people

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

Expected to start

Aug 2026

An estimate. Start dates often move.

Expected to finish

Jul 2030

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

18 to 55 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. Aged 18 to 55 years, regardless of gender. 2. Comprehensive clinical, biochemical, and genetic diagnosis of hereditary homocysteine remethylation disorders. 3. Evidence of neurological involvement, including but not limited to gait disturbance, balance impairment, cognitive dysfunction, cerebral white matter lesions. 4. Prior standardized metabolic therapy (folic acid, vitamin B12, betaine) with suboptimal clinical response. 5. Persistent severe metabolic abnormality, i.e., sustained elevated homocysteine (\>50 umol/L). 6. Multidisciplinary consensus confirming lack of effective alternative therapies and ongoing risk of disease progression. 7. Voluntary participation, signed informed consent, adequate treatment adherence, and willingness to complete follow-up assessments. Exclusion Criteria: 1. Prior hematopoietic stem cell transplantation or other cell transplantation. 2. Severe dysfunction of critical organs (heart, lung, liver, kidney) deemed incompatible with study treatment by investigators. 3. Active, uncontrolled infection. 4. Active tuberculosis, hepatitis B, hepatitis C, HIV infection, or other infectious diseases judged inappropriate for enrollment by investigators. 5. Active malignancy or prior malignant history that may confound safety and efficacy evaluations. 6. Severe underlying comorbidities likely to interfere with study treatment or outcome assessment. 7. Severe psychiatric disorder or cognitive impairment with poor adherence precluding completion of treatment and follow-up. 8. Pregnant or lactating females, or participants unwilling to use effective contraception throughout the study period. 9. Severe hypersensitivity to any study-related medication or intervention. 10. Participation in other interventional clinical trials within the past 4 weeks or ongoing observation period of another clinical trial. 11. No documented disease progression within the preceding 12 months. 12. Minimal neurological symptoms with no meaningful impact on activities of daily living and low short-term progression risk per investigator assessment. 13. Established standard therapies proven to alter natural disease history with stable disease and satisfactory therapeutic response. 14. End-stage disease with extensive irreversible neurological impairment (severe motor/cognitive failure or multi-organ dysfunction) with minimal expected therapeutic benefit. 15. Any other conditions deemed unsuitable for study participation by investigators.

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As listed by the trial registrant

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

How to take part

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

  1. The places running it

    2 sites. 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

  • General Hospital of Tianjin Medical University

    Tianjin, Tianjin Municipality, 300070, China

  • Institute of Hematology & Blood Diseases Hospital, China

    Tianjin, Tianjin Municipality, 300020, China