Immune Thrombocytopenia (ITP)Phase 2PKU-ITP-FMTNCT07733037
Fecal Microbiota Transplantation for Steroid-resistant or Relapsed Immune Thrombocytopenia
A Phase 2 study testing Fecal microbiota transplantation in people with Immune Thrombocytopenia (ITP), run by Peking University People's Hospital at 1 site in China.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Immune Thrombocytopenia (ITP)
- What is being tested?
- Fecal microbiota transplantation
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 1 siteChina
- Study dates
- 10 Jan 2026to 30 Sep 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Fecal Microbiota Transplantation for Patients With Steroid-Resistant or Relapsed Immune Thrombocytopenia: A Randomized, Double-blind, Placebo-Controlled Phase 2 Trial
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
Read the 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.
- Conditions
- Immune Thrombocytopenia (ITP)
- Being tested
- Fecal microbiota transplantation
- Keywords
- Immune thrombocytopenia, Fecal microbiota transplantation
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal microbiota transplantationDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Fecal microbiota transplantation group | Experimental | Patients in the fecal microbiota transplantation group receive bowel preparation and standard fecal microbiota capsules for 6 days Receives: Drug: Fecal microbiota transplantation |
| Placebo group | Placebo comparator | Patients in the placebo group receive bowel preparation and standard placebo capsules for 6 days Receives: Drug: Fecal microbiota transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 12 (±7 days) after the initiation of treatment
Secondary outcomes (6)
Complete Response Rate (CR)
The proportion of subjects whose platelet count reaches or exceeds 100×10⁹/L at any time point after treatment.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 27 months
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.
Measured: 27 months
Adverse events
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version5.0.
Measured: 27 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2026
- Study start10 Jan 2026
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients are randomly assigned at a 2:1 ratio to receive fecal microbiota transplantation or placebo.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07733037
- Sponsor's protocol ID
- 2025PHB534-001
- Acronym
- PKU-ITP-FMT
- Status last verified
- Jul 2026
- First submitted
- 23 Jul 2026
- Last updated
- 29 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fecal Microbiota Transplantation for Patients With Steroid-Resistant or Relapsed Immune Thrombocytopenia: A Randomized, Double-blind, Placebo-Controlled Phase 2 Trial
- ClinicalTrials.gov ID
- NCT07733037
- Acronym / sponsor ID
- PKU-ITP-FMT / 2025PHB534-001
- Conditions
- Immune Thrombocytopenia (ITP)
- Keywords
- Immune thrombocytopenia, Fecal microbiota transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 10 Jan 2026 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 29 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Patients are randomly assigned at a 2:1 ratio to receive fecal microbiota transplantation or placebo.
| Arm | Type | Receives |
|---|---|---|
| Fecal microbiota transplantation group | Experimental | Drug: Fecal microbiota transplantationPatients in the fecal microbiota transplantation group receive bowel preparation and standard fecal microbiota capsules for 6 days |
| Placebo group | Placebo comparator | Drug: Fecal microbiota transplantationPatients in the placebo group receive bowel preparation and standard placebo capsules for 6 days |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fecal microbiota transplantationPatients 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. | Drug | Fecal microbiota transplantation group, Placebo group |
Primary outcome
Overall Response Rate (ORR) at Week 12 of Treatment
Time frame: 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.
6 secondary outcomes
Complete Response Rate (CR)
Time frame: 27 months
Duration of Sustained Response
Time frame: 27 months
Bleeding Score
Time frame: 27 months
Bleeding events
Time frame: 27 months
Health-related quality of life (HRQOL)
Time frame: 27 months
Adverse events
Time frame: 27 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fecal microbiota transplantation in people with Immune Thrombocytopenia (ITP), run by Peking University People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 29 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) at Week 12 of Treatment (time frame: Week 12 (±7 days) after the initiation of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2026.