Can a Patient's own stem cells reset the immune system to halt brain damage?
NCT ID NCT07757685
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
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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
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About 50 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Jul 2030
An estimate. End dates often move.
- Lead sponsor
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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
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18 to 55 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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General Hospital of Tianjin Medical University
Tianjin, Tianjin Municipality, 300070, China
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Institute of Hematology & Blood Diseases Hospital, China
Tianjin, Tianjin Municipality, 300020, China