Multi-target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia

August 24, 2026 updated by: Institute of Hematology & Blood Diseases Hospital, China

Efficacy and Safety of Multi-Target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia: A Prospective, Single-arm, Multicenter, Exploratory Trial

This prospective, single-arm, multicenter, exploratory study will enroll 38 adults patients with newly diagnosed primary immune thrombocytopenia (ITP). Patients will receive standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA) as initial therapy During the core treatment phase (Weeks 1-12). Corticosteroids will be tapered and discontinued within 8 weeks, whereas TPO-RA treatment will continue for 12 weeks. For patients with treatment failure( defined as platelet count < 30 × 10^9/L or less than 2-fold increase of baseline platelet count or bleeding) ,a sequential multitarget combination strategy will be explored in subsequent treatment phases. Specifically, patients with treatment failure after 2 weeks of initial therapy (Weeks 3-12), will receive the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody（mAb） as sequential combination therapy.

During the core treatment follow-up phase (24 weeks), patients with treatment failure will enter the exploratory treatment phase (Weeks 13-36) .

Patients who received multi-target drug therapy during the core treatment period will switch to an alternative TPO-RA with cross-administered rituximab and anti-CD38 mAb, while those who did not will receive sequential rituximab or anti-CD38 mAb.

Finally, patients will enter the safety follow up period (4 weeks, weeks 37-40).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The study will be conducted in three phases.

Phase 1. Core treatment phase includes initial treatment and sequential combination therapy (12 weeks, weeks 1-12):

Patients will receive standard-dose methylprednisolone or prednisone in combination with a thrombopoietin receptor agonist (TPO-RA) as initial therapy.Corticosteroids will be tapered and discontinued within 8 weeks; tapering will begin immediately in patients who achieve complete response (CR) and will also be initiated after 4 weeks in patients who do not achieve response (R). TPO-RA therapy will continue for 12 weeks, with dose adjustments made according to the approved prescribing information. Patients with treatment failure( defined as platelet count < 30 × 10^9/L or less than 2-fold increase of baseline platelet count or bleeding) after 2 weeks of initial therapy (Weeks 3-12) will receive the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody as sequential combination therapy.

Phase 2. Core-treatment follow-up and exploratory treatment phase (24weeks, weeks 13-36):

During the core treatment follow-up phase, patients with treatment failure will enter the exploratory treatment phase. Patients who did not receive rituximab or anti-CD38 monoclonal antibody during the core treatment phase will receive a switched TPO-RA combined with either rituximab or anti-CD38 monoclonal antibody, whereas patients who received either rituximab or anti-CD38 monoclonal antibody during the core phase will receive a switched TPO-RA combined with cross-administered rituximab and anti-CD38 monoclonal antibody.TPO-RA therapy will also continue for 12 weeks.

Phase3. Safety follow-up phase (4weeks,weeks 37-40).

Rescue therapy includes, but is not limited to, intravenous immunoglobulin (IVIG), platelet transfusion, and vindesine. A switch to another thrombopoietin receptor agonist (TPO-RA) will be considered rescue therapy during the core treatment period. Administration of recombinant human thrombopoietin (rhTPO) will also be considered rescue therapy.

Study Type

Interventional

Enrollment (Estimated)

38

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
      • Tianjin, China, 300020
        • Recruiting
        • Ethics Committee of Hematology Hospital, Chinese Academy of Medical Sciences
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age ≥18 years.
  2. Confirmed newly diagnosed primary ITP with a platelet count <30 × 10^9/L.
  3. No prior ITP-related treatment before enrollment, except for standard-dose corticosteroids and/or IVIG for ≤5 days.
  4. Able to understand the study and provide signed informed consent.-

Exclusion Criteria:

  1. Use of corticosteroids or immunosuppressants for a non-ITP condition within 3 months.
  2. Contraindication to corticosteroid therapy.
  3. Arterial or venous thromboembolic event within 3 months.
  4. Pregnant or breastfeeding women.
  5. Current treatment with another investigational drug.

  6. Any other medical history or condition that, in the investigator's judgment, makes the participant unsuitable for the study.

    -

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Initial therapy
standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA)
Drug: standard-dose methylprednisolone/prednisone.
Methylprednisolone 0.8-1mg/kg/day administered intravenously or orally; or prednisone 1 mg/kg/day, up to a maximum dose of 80 mg/day
Drug: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.
Dosing will follow the recommended dose in the prescribing information; in severe ITP, the initial dose will be selected according to BAT (best available therapy) principles.
Experimental: Sequential Combination Therapy
the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody（mAb）
Drug: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.
Dosing will follow the recommended dose in the prescribing information; in severe ITP, the initial dose will be selected according to BAT (best available therapy) principles.
Drug: rituximab.
Administered as 375 mg/m² by intravenous infusion for a single dose, or 100 mg by intravenous infusion once weekly for a total of 4 doses.
Drug: Daratumumab
Administered at 16 mg/kg by intravenous infusion once weekly for a total of 4-8 doses.
Experimental: Exploratory treatment
a switched TPO-RA combined with either rituximab or anti-CD38 monoclonal antibody
Drug: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.
Dosing will follow the recommended dose in the prescribing information; in severe ITP, the initial dose will be selected according to BAT (best available therapy) principles.
Drug: rituximab.
Administered as 375 mg/m² by intravenous infusion for a single dose, or 100 mg by intravenous infusion once weekly for a total of 4 doses.
Drug: Daratumumab
Administered at 16 mg/kg by intravenous infusion once weekly for a total of 4-8 doses.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
sustained response rate
Time Frame: between weeks 13 and 36
Defined as the proportion of patients who achieve a platelet count ≥30×10^9/L at least 6 out of 12 scheduled visits during the 24 weeks following the core treatment period (weeks 13-36), in the absence of rescue therapy
between weeks 13 and 36

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
time to response
Time Frame: in 0-12 weeks
The time from treatment initiation to achieve a complete response(platelet count ≥100 × 109/L and absence of bleeding) or a partial response(platelet count ≥30 × 10^9/L and at least 2-fold increase of the baseline platelet count and absence of bleeding)
in 0-12 weeks
Early response.
Time Frame: at 1 week
Proportion of patients achieving platelet count ≥30 × 10^9/L and at least doubling baseline at 1 week after treatment initiation.
at 1 week
Initial response
Time Frame: at 1 month
Proportion of patients achieving platelet count ≥30 × 10^9/L and at least doubling baseline at 1 month after treatment initiation.
at 1 month
overall response(OR) rate at week 12
Time Frame: at week 12
Proportion of patients achieving complete response (CR) plus response (R) at Week 12
at week 12
Proportion of patients requiring rescue therapy
Time Frame: in 0-36 weeks
in 0-36 weeks
bleeding scores
Time Frame: in 0-40 weeks
Assessed according to the WHO Bleeding Scale and the ITP Bleeding Scale
in 0-40 weeks
Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
Time Frame: in 0-40 weeks
In all participants ,use ITP-PAQ to assess the HRQoL before and after treatment.
in 0-40 weeks
Functional Assessment of 36-Item Short Form Survey (SF-36) scale
Time Frame: in 0-40 weeks
In all participants ,use SF-36 scale to assess the HRQoL before and after treatment.
in 0-40 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Investigators

  • Study Director: Lei Zhang, Thrombosis and Haemostasis Diagnosis Treatment Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 22, 2026

Primary Completion (Estimated)

May 30, 2027

Study Completion (Estimated)

June 30, 2027

Study Registration Dates

First Submitted

August 5, 2026

First Submitted That Met QC Criteria

August 24, 2026

First Posted (Actual)

August 26, 2026

Study Record Updates

Last Update Posted (Actual)

August 26, 2026

Last Update Submitted That Met QC Criteria

August 24, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IIT2025106
  • Investigator-Initiated Trial (Other Grant/Funding Number: Chinese Academy of Medical Sciences (CAMS) Innovation Fund for Medical Sciences)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

No plan to share IPD has been established at this time

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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