This is a randomized, double-blind, placebo-controlled phase 2 trial to evaluate the safety and efficacy in immune thrombocytopenia patients receiving fecal microbiota transplantation
This is a prospective, randomized, double-blind, placebo-controlled design of 42 adult patients with steroid-resistantrelapse ITP in China. Patients are randomly assigned at a 2:1 ratio to receive fecal microbiota transplantation or placebo. Patients in the fecal microbiota transplantation group receive bowel preparation and standard fecal microbiota capsules for 6 days. Those in the placebo group receive bowel preparation and standard placebo capsules for 6 days. Treatment wil be discontinued if very severe or life-threateningadverse events developed or at the patients' request. The primary endpoint is the overall response rate (ORR) at week 12 of treatment, defined as the rate of patients who obtain a platelet count \> 50,000/uL, and at least 30,000/uL increase of the baseline count.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
42
Patients in the fecal microbiota transplantation group receive bowel preparation and standard fecal microbiota capsules for 6 days. Those in the placebo group receive bowel preparation and standard placebo capsules for 6 days. Treatment wil be discontinued if very severe or life-threateningadverse events developed or at the patients' request.
Peking University People's Hospital
Beijing, China
Overall Response Rate (ORR) at Week 12 of Treatment
the rate of patients who obtain a platelet count \> 50,000/uL, and at least 30,000/uL increase of the baseline count at week 12 (±7 days) after the initiation of treatment.
Time frame: Week 12 (±7 days) after the initiation of treatment
Complete Response Rate (CR)
The proportion of subjects whose platelet count reaches or exceeds 100×10⁹/L at any time point after treatment.
Time frame: 27 months
Duration of Sustained Response
The time interval from the first achievement of the criteria of platelet count reached or exceeded 50×10⁹/L and increased by at least 30×10⁹/L from baseline simultaneously until the occurrence of platelet count relapse (falling below the criteria) or the requirement for additional ITP treatment.
Time frame: 27 months
Bleeding Score
The internationally standardized Immune Thrombocytopenia Bleeding Assessment Tool (ITP-BAT) was used for scoring, and the changes in scores at each follow-up visit compared with baseline were analyzed. Bleeding symptoms are grouped into three major domains: skin (S), visible mucosae (M), and organ (and internal mucosae) (O). Grading ranges from 0 to 4 for epistaxis and for bleeding in the organ domain, except ocular and intracranial bleeding (grade 0 and 2 to 4). For the remaining bleeding sites (in skin and mucosal domains) four grades (0 to 3) were deemed sufficient. Grade 5 is assigned to any fatal bleeding.
Time frame: 27 months
Bleeding events
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale: 0, no bleeding; 1, petechiae; 2, mild blood loss; 3, gross blood loss; and 4, debilitating blood loss.
Time frame: 27 months
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Health-related quality of life (HRQOL)
ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment. The ITP-PAQ is a 44-item disease-specific questionnaire for ITP, covering ten independent scales including Symptoms, Fatigue/Sleep, Women's Reproductive Health and Overall QoL to comprehensively measure disease-related health quality of life. Each scale of ITP-PAQ adopts a 0-100 scoring system, and higher scores indicate better health-related quality of life among ITP patients.
Time frame: 27 months
Adverse events
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version5.0.
Time frame: 27 months