ITP - Immune ThrombocytopeniaPhase 4NCT07788456
Multi-target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia
A Phase 4 study testing standard-dose methylprednisolone/prednisone., thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag. and rituximab. in people with ITP - Immune Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- ITP - Immune Thrombocytopenia
- What is being tested?
- standard-dose methylprednisolone/prednisone., thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag. and rituximab.
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteChina
- Study dates
- 22 Jun 2026to 30 Jun 2027
- Phase
- Phase 4
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.-
Who cannot join
- 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.
- -
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Rongfeng Fu contact
China1 site, 1 recruiting
- Recruiting
Ethics Committee of Hematology Hospital, Chinese Academy of Medical Sciences
Tianjin, 300020
Shuo Chen, +86-022-23608025, ec@ihcams.ac.cn
About this study
Official title: 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) .
Read more
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).
Read the 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.
- Conditions
- ITP - Immune Thrombocytopenia
- Being tested
- standard-dose methylprednisolone/prednisone., thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag., rituximab., Daratumumab
- Keywords
- corticosteroid, thrombopoietin receptor agonist （TPO-RA）
- 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.
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
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.
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.
DaratumumabDrug
Administered at 16 mg/kg by intravenous infusion once weekly for a total of 4-8 doses.
| Study group | Role | What they receive |
|---|---|---|
| Initial therapy | Experimental | standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA) Receives: 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） Receives: 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 Receives: Drug: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.; Drug: rituximab.; Drug: Daratumumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: between weeks 13 and 36
Secondary outcomes (8)
time to response
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)
Measured: in 0-12 weeks
Early response.
Proportion of patients achieving platelet count ≥30 × 10^9/L and at least doubling baseline at 1 week after treatment initiation.
Measured: at 1 week
Initial response
Proportion of patients achieving platelet count ≥30 × 10^9/L and at least doubling baseline at 1 month after treatment initiation.
Measured: at 1 month
overall response(OR) rate at week 12
Proportion of patients achieving complete response (CR) plus response (R) at Week 12
Measured: at week 12
Proportion of patients requiring rescue therapy
Measured: in 0-36 weeks
bleeding scores
Assessed according to the WHO Bleeding Scale and the ITP Bleeding Scale
Measured: in 0-40 weeks
Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
In all participants ,use ITP-PAQ to assess the HRQoL before and after treatment.
Measured: in 0-40 weeks
Functional Assessment of 36-Item Short Form Survey (SF-36) scale
In all participants ,use SF-36 scale to assess the HRQoL before and after treatment.
Measured: in 0-40 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2026
- Study start22 Jun 2026
- Primary completion30 May 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for ITP - Immune ThrombocytopeniaStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, Study director, Thrombosis and Haemostasis Diagnosis Treatment Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07788456
- Sponsor's protocol ID
- IIT2025106
- Other IDs
- Investigator-Initiated Trial
- Status last verified
- Jul 2026
- First submitted
- 5 Aug 2026
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No plan to share IPD has been established at this time
Publications
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.
PubMed 23293082Chen 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.
PubMed 38899695Rodeghiero 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.
PubMed 19005182Provan 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.
PubMed 31770441Neunert 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.
PubMed 31794604