Real-World Study on Fecal Microbiota Transplantation: Long-Term Effectiveness and Safety Statistics
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 4,000
- 试验地点
- 1
- 主要终点
- 8-Week Clinical Cure of Recurrent CDI
研究概览
简要总结
This is a large-scale observational study aiming to evaluate the long-term effectiveness and safety of Fecal Microbiota Transplantation (FMT). FMT is a procedure that transfers gut bacteria from healthy donors to patients to restore a balanced gut microbiome.
The study will follow approximately 4,000 patients who have received or will receive FMT for conditions like recurrent C. difficile infection, inflammatory bowel disease, functional gastrointestinal disorders, and certain neurological conditions.
The main goals are to:
- Assess the disease remission rates at 3 months, 1 year, and 5 years after FMT.
- Monitor the long-term safety and any potential side effects.
- Identify factors that may influence how well a patient responds to the treatment.
This research will use both existing patient data (retrospective cohort) and newly collected data from future patients (prospective cohort). The findings are expected to help improve and standardize FMT treatment for better patient care.
详细描述
This is a single-center, real-world observational study combining retrospective and prospective cohorts to systematically evaluate the long-term effectiveness and safety of Fecal Microbiota Transplantation (FMT).
The study plans to enroll approximately 4,000 patients who have undergone FMT for a range of diseases, including recurrent Clostridium difficile infection, inflammatory bowel disease, functional gastrointestinal diseases, and certain extra-intestinal disorders.
The primary objective is to assess the real-world clinical remission rates at 3 months, 1 year, and 5 years post-FMT. Secondary objectives include evaluating long-term safety and adverse events, measuring improvements in quality of life, and exploring factors (such as delivery route, donor selection, and patient characteristics) that predict treatment success. Data will be collected from medical records and through planned follow-up visits. Advanced statistical and machine learning models will be used to analyze the data and build predictive models.
The results of this large-scale study will provide high-quality evidence to support the standardized and precise clinical application of FMT.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with one of the target diseases.
- •Age ≥ 3 years.
- •Have received at least one Fecal Microbiota Transplantation (FMT) treatment.
- •Availability of complete baseline and follow-up data for analysis.
- •Provide signed informed consent (for the prospective cohort) OR consent for the use of clinical data (for the retrospective cohort).
排除标准
- •Clinical data is severely missing, making efficacy assessment impossible.
- •Presence of severe complications that may jeopardize safety or confound the study results.
结局指标
主要结局
8-Week Clinical Cure of Recurrent CDI
时间窗: 8 weeks after the final FMT treatment
Percentage of participants with sustained clinical cure at 8 weeks post-treatment, defined as persistent resolution of diarrhea (formed stools \<3 times per day) AND a negative test for C. difficile toxin.
Treatment Response in Chronic Constipation at 8 Weeks
时间窗: 8 weeks after the final FMT treatment
Percentage of participants meeting all of the following criteria at 8 weeks: \>3 complete spontaneous bowel movements per week, Bristol Stool Form Scale (BSFS) type 3-5, and a reduction of \>30% in the Patient Assessment of Constipation Symptoms (PAC-SYM) score.
Corticosteroid-Free Clinical Remission in IBD at 8 Weeks
时间窗: 8 weeks after the final FMT treatment
Percentage of participants achieving corticosteroid-free clinical remission. For Ulcerative Colitis (UC), defined as a Mayo score ≤2 with no subscore \>1. For Crohn's Disease (CD), defined as a Crohn's Disease Activity Index (CDAI) score \<150.
Symptom Relief in IBS at 3 Months
时间窗: 3 months after the final FMT treatment
Percentage of participants experiencing relief of abdominal pain or discomfort related to defecation for 3 consecutive months, with stool consistency rated as Bristol Stool Form Scale (BSFS) type 3-5.
Symptom Resolution in Drug-Induced Enteritis
时间窗: 8 weeks after the final FMT treatment
Percentage of participants achieving symptom resolution, defined as a reduction to CTCAE grade ≤1 (stool frequency increase \<4 times per day from baseline, without abdominal pain, hematochezia, or mucus), AND requiring corticosteroids \<10 mg prednisone equivalent per day.
Treatment Response in Autism at 12 Weeks
时间窗: 12 weeks after the final FMT treatment
Percentage of participants meeting the response criteria at Week 12, defined as an Autism Behavior Checklist (ABC) score \<31 AND a score of 1 (very much improved) on the Clinical Global Impression-Improvement (CGI-I) scale.
Symptom Improvement in Incomplete Bowel Obstruction
时间窗: 8 weeks after the final FMT treatment
Percentage of participants achieving a reduction of \>50% in abdominal distension and/or pain, AND weaning off parenteral nutrition.
次要结局
未报告次要终点
研究者
Chen QiYi
Chief of Functional Gastrointestinal Surgery
Shanghai 10th People's Hospital