Immune Thrombocytopenia (ITP)
Conditions
Keywords
Immune thrombocytopenia, Fecal microbiota transplantation
Brief summary
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
Detailed description
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.
Interventions
DRUGFecal microbiota transplantation
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.
Sponsors
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
This is a prospective, randomized, double-blind, placebo-controlled trial.
Intervention model description
Patients are randomly assigned at a 2:1 ratio to receive fecal microbiota transplantation or placebo.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Aged 18 to 70 years, regardless of gender. 2. Patients diagnosed with immune thrombocytopenia (ITP) in line with the diagnostic criteria, and with a disease course of ≥ 12 months (chronic ITP). 3. Meeting the definitions of corticosteroid resistance or relapse (resistance: platelet count remains \< 30×10⁹/L despite treatment with standard-dose corticosteroids for ≥ 4 weeks; relapse: platelet count drops again to \< 30×10⁹/L after corticosteroid dose reduction or discontinuation). 4. Sustained platelet count \< 30×10⁹/L during the screening period. 5. Subjects voluntarily participate in this study and sign a written informed consent form.
Exclusion criteria
1. Secondary thrombocytopenia caused by other factors, such as drug-induced etiology, infection-related conditions, or other autoimmune diseases (e.g., systemic lupus erythematosus). 2. Subjects positive for Helicobacter pylori (H. pylori) infection (rescreening is permitted only after completion of standard eradication therapy and a drug withdrawal period of at least 1 month). 3. Active infection or receipt of systemic antibiotic therapy within the past 4 weeks. 4. Severe cardiac, hepatic, or renal insufficiency (e.g., Child-Pugh Class C liver dysfunction, or estimated glomerular filtration rate \< 30 mL/min/1.73 m²). 5. A history of malignant tumors or primary immunodeficiency diseases. 6. Pregnant or lactating women. 7. Previous splenectomy, rituximab therapy, or plasma exchange treatment within the past 3 months. 8. Other conditions deemed inappropriate for study participation by the investigators based on clinical judgment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Response Rate (ORR) at Week 12 of Treatment | Week 12 (±7 days) after the initiation 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete Response Rate (CR) | 27 months | The proportion of subjects whose platelet count reaches or exceeds 100×10⁹/L at any time point after treatment. |
| Duration of Sustained Response | 27 months | 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. |
| Bleeding Score | 27 months | 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. |
| Bleeding events | 27 months | 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. |
| Health-related quality of life (HRQOL) | 27 months | 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. |
| Adverse events | 27 months | Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version5.0. |
Countries
China
Outcome results
None listed