Autologous Hematopoietic Stem Cell Transplantation for Neurological Damage Associated With Heredi… (NCT07757685) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Autologous Hematopoietic Stem Cell Transplantation for Neurological Damage Associated With Hereditary Homocysteine Remethylation Disorders
China50 participantsStarted 2026-08-01
Plain-language summary
This study aims to evaluate the safety, feasibility, and preliminary efficacy of autologous hematopoietic stem cell transplantation (ASCT) in the treatment of neurological damage associated with hereditary homocysteine remethylation disorders. Meanwhile, peripheral blood, cerebrospinal fluid, and related clinical samples will be prospectively collected before and after transplantation to dynamically monitor changes in immune reconstitution and neuroinflammatory biomarkers. The study intends to explore the impact of immune system resetting on disease progression and central nervous system immune microenvironment, providing evidence for subsequent precise patient stratification and optimized therapeutic strategies.
Who can participate
Age range
18 Years – 55 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Aged 18 to 55 years, regardless of gender.
. Comprehensive clinical, biochemical, and genetic diagnosis of hereditary homocysteine remethylation disorders.
. Evidence of neurological involvement, including but not limited to gait disturbance, balance impairment, cognitive dysfunction, cerebral white matter lesions.
. Multidisciplinary consensus confirming lack of effective alternative therapies and ongoing risk of disease progression.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Primary Endpoint: Incidence of adverse events within 100 days post ASCT
Timeframe: 100 days post ASCT
Trial details
NCT IDNCT07757685
SponsorInstitute of Hematology & Blood Diseases Hospital, China