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

NCT07788456 | Recruiting Phase 4 DMC

• 2 other identifiers: IIT2025106Investigator-Initiated Trial
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

ITP - Immune Thrombocytopenia

Keywords

corticosteroid; thrombopoietin receptor agonist （TPO-RA）

Outcome Measures

Primary Outcomes (1)

• sustained response rate

  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 Outcomes (8)

• time to response

  in 0-12 weeks

• Early response.

  at 1 week

• Initial response

  at 1 month

• overall response(OR) rate at week 12

  at week 12

• Proportion of patients requiring rescue therapy

  in 0-36 weeks

Study Arms (3)

Initial therapy

EXPERIMENTAL

standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA)

Drug: standard-dose methylprednisolone/prednisone.; Drug: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.

Sequential Combination Therapy

EXPERIMENTAL

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.; Drug: rituximab.; Drug: Daratumumab

Exploratory treatment

EXPERIMENTAL

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.; Drug: rituximab.; Drug: Daratumumab

Interventions

standard-dose methylprednisolone/prednisone. DRUG

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

Initial therapy

rituximab. DRUG

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.

Exploratory treatment; Sequential Combination Therapy

Daratumumab DRUG

Administered at 16 mg/kg by intravenous infusion once weekly for a total of 4-8 doses.

Exploratory treatment; Sequential Combination Therapy

thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag. DRUG

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.

Exploratory treatment; Initial therapy; Sequential Combination Therapy

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Use of corticosteroids or immunosuppressants for a non-ITP condition within 3 months.
• Contraindication to corticosteroid therapy.
• Arterial or venous thromboembolic event within 3 months.
• Pregnant or breastfeeding women.
• Current treatment with another investigational drug.
• Any other medical history or condition that, in the investigator's judgment, makes the participant unsuitable for the study.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Ethics Committee of Hematology Hospital, Chinese Academy of Medical Sciences

Tianjin, 300020, China

RECRUITING

Related Publications (5)

• Gudbrandsdottir S, Birgens HS, Frederiksen H, Jensen BA, Jensen MK, Kjeldsen L, Klausen TW, Larsen H, Mourits-Andersen HT, Nielsen CH, Nielsen OJ, Plesner T, Pulczynski S, Rasmussen IH, Ronnov-Jessen D, Hasselbalch HC. Rituximab and dexamethasone vs dexamethasone monotherapy in newly diagnosed patients with primary immune thrombocytopenia. Blood. 2013 Mar 14;121(11):1976-81. doi: 10.1182/blood-2012-09-455691. Epub 2013 Jan 4.

  PMID: 23293082 BACKGROUND

• Chen Y, Xu Y, Li H, Sun T, Cao X, Wang Y, Xue F, Liu W, Liu X, Dong H, Fu R, Dai X, Wang W, Ma Y, Song Z, Chi Y, Ju M, Gu W, Pei X, Yang R, Zhang L. A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia. N Engl J Med. 2024 Jun 20;390(23):2178-2190. doi: 10.1056/NEJMoa2400409.

  PMID: 38899695 BACKGROUND

• Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.

  PMID: 19005182 BACKGROUND

• Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, Gonzalez-Lopez TJ, Grainger J, Hou M, Kruse C, McDonald V, Michel M, Newland AC, Pavord S, Rodeghiero F, Scully M, Tomiyama Y, Wong RS, Zaja F, Kuter DJ. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817. doi: 10.1182/bloodadvances.2019000812.

  PMID: 31770441 BACKGROUND

• Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966.

  PMID: 31794604 BACKGROUND

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Methylprednisolone; Prednisone; eltrombopag; Rituximab; daratumumab

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)

Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Pregnadienediols; Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Lei Zhang

  Thrombosis and Haemostasis Diagnosis Treatment Center

  STUDY DIRECTOR

Central Study Contacts

Xiaofan Liu

CONTACT

+86-022-23608180; liuxiaofan@ihcams.ac.cn

Rongfeng Fu

CONTACT

furongfeng@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2026
• First Posted: August 26, 2026
• Study Start: June 22, 2026
• Primary Completion (Estimated): May 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: August 26, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)