Fecal Microbiota Transplantation for Steroid-resistant or Relapsed Immune Thrombocytopenia

NCT07733037 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: 2025PHB534-001
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

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

Conditions

Immune Thrombocytopenia (ITP)

Keywords

Immune thrombocytopenia; Fecal microbiota transplantation

Outcome Measures

Primary Outcomes (1)

• 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.

  Week 12 (±7 days) after the initiation of treatment

Secondary Outcomes (6)

• Complete Response Rate (CR)

  27 months

• Duration of Sustained Response

  27 months

• Bleeding Score

  27 months

• Bleeding events

  27 months

• Health-related quality of life (HRQOL)

  27 months

Other Outcomes (2)

• Alterations in the Fecal Microbiota

  Screening period, Week 12 (±7), Week 36 (±7)

• Alterations in immune profiles

  Screening period, Week 12 (±7), Week 36 (±7)

Study Arms (2)

Fecal microbiota transplantation group

EXPERIMENTAL

Patients in the fecal microbiota transplantation group receive bowel preparation and standard fecal microbiota capsules for 6 days

Drug: Fecal microbiota transplantation

Placebo group

PLACEBO COMPARATOR

Patients in the placebo group receive bowel preparation and standard placebo capsules for 6 days

Drug: Fecal microbiota transplantation

Interventions

Fecal microbiota transplantation DRUG

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.

Fecal microbiota transplantation group; Placebo group

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 18 to 70 years, regardless of gender.
• Patients diagnosed with immune thrombocytopenia (ITP) in line with the diagnostic criteria, and with a disease course of ≥ 12 months (chronic ITP).
• 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).
• Sustained platelet count \< 30×10⁹/L during the screening period.
• Subjects voluntarily participate in this study and sign a written informed consent form.

You may not qualify if:

• Secondary thrombocytopenia caused by other factors, such as drug-induced etiology, infection-related conditions, or other autoimmune diseases (e.g., systemic lupus erythematosus).
• 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).
• Active infection or receipt of systemic antibiotic therapy within the past 4 weeks.
• 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).
• A history of malignant tumors or primary immunodeficiency diseases.
• Pregnant or lactating women.
• Previous splenectomy, rituximab therapy, or plasma exchange treatment within the past 3 months.
• Other conditions deemed inappropriate for study participation by the investigators based on clinical judgment.

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University People's Hospital

Beijing, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This is a prospective, randomized, double-blind, placebo-controlled trial.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking University Institute of Hematology

Model Details: Patients are randomly assigned at a 2:1 ratio to receive fecal microbiota transplantation or placebo.

Study Record Dates

• First Submitted: July 23, 2026
• First Posted: July 29, 2026
• Study Start: January 10, 2026
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): September 30, 2028
• Last Updated: July 29, 2026

Record last verified: 2026-07

Locations

CN (1)