NCT07757685尚未招募不适用
Exploratory Clinical Study of Autologous Hematopoietic Stem Cell Transplantation for Neurological Damage Associated With Hereditary Homocysteine Remethylation Disorders
Institute of Hematology & Blood Diseases Hospital, China2 个研究点 分布在 1 个国家目标入组 50 人开始时间: 2026年8月1日最近更新:
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 50
- 试验地点
- 2
- 主要终点
- Primary Endpoint: Incidence of adverse events within 100 days post ASCT
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
- •Prior standardized metabolic therapy (folic acid, vitamin B12, betaine) with suboptimal clinical response.
- •Persistent severe metabolic abnormality, i.e., sustained elevated homocysteine (>50 umol/L).
- •Multidisciplinary consensus confirming lack of effective alternative therapies and ongoing risk of disease progression.
- •Voluntary participation, signed informed consent, adequate treatment adherence, and willingness to complete follow-up assessments.
排除标准
- •Prior hematopoietic stem cell transplantation or other cell transplantation.
- •Severe dysfunction of critical organs (heart, lung, liver, kidney) deemed incompatible with study treatment by investigators.
- •Active, uncontrolled infection.
- •Active tuberculosis, hepatitis B, hepatitis C, HIV infection, or other infectious diseases judged inappropriate for enrollment by investigators.
- •Active malignancy or prior malignant history that may confound safety and efficacy evaluations.
- •Severe underlying comorbidities likely to interfere with study treatment or outcome assessment.
- •Severe psychiatric disorder or cognitive impairment with poor adherence precluding completion of treatment and follow-up.
- •Pregnant or lactating females, or participants unwilling to use effective contraception throughout the study period.
- •Severe hypersensitivity to any study-related medication or intervention.
- •Participation in other interventional clinical trials within the past 4 weeks or ongoing observation period of another clinical trial.
- •No documented disease progression within the preceding 12 months.
- •Minimal neurological symptoms with no meaningful impact on activities of daily living and low short-term progression risk per investigator assessment.
- •Established standard therapies proven to alter natural disease history with stable disease and satisfactory therapeutic response.
- •End-stage disease with extensive irreversible neurological impairment (severe motor/cognitive failure or multi-organ dysfunction) with minimal expected therapeutic benefit.
- •Any other conditions deemed unsuitable for study participation by investigators.
研究组 & 干预措施
Patients comprehensively diagnosed with hereditary homocysteine remethylation disorders with neurolo
Experimental
干预措施: Autologous Hematopoietic Stem Cell Transplantation (Other)
结局指标
主要结局
Primary Endpoint: Incidence of adverse events within 100 days post ASCT
时间窗: 100 days post ASCT
次要结局
- Time to platelet engraftment(1 year post ASCT)
- Success rate of autologous hematopoietic stem cell collection(Within 3 days after initiation of stem cell collection)
- Time to neutrophil engraftment(28 days post ASCT)
- 1-year overall survival post ASCT(1-year post ASCT)
- Changes in neurological functional scores(from baseline to 1 year after ASCT)
- Changes in MRI lesions(from baseline to 1 year after ASCT)
- PET-CT findings(from baseline to 1 year after ASCT)
- Kinetics of peripheral immune reconstitution(from baseline to 1 year after ASCT)
- Changes in inflammatory and neuronal injury biomarkers(from baseline to 1 year after ASCT)
研究者
研究点 (2)
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