Primary Immune Thrombocytopenia (ITP)Phase 4NCT07189910
Sequential rhTPO and Eltrombopag Following Glucocorticoids for Severe Adult ITP
A Phase 4 study testing Eltrombopag in people with Primary Immune Thrombocytopenia (ITP), run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology at 1 site in China.
Trial summary
To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2025to 31 Dec 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 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age between 12 and 75 years, regardless of gender;
- ECOG performance status of 0-1;
- Diagnosis of primary ITP confirmed by bone marrow biopsy (valid within 3 months) and other relevant assessments; baseline platelet count (PLT) <20×10⁹/L within 48 hours prior to administration of study medication;
- Adequate function of major organs as defined below (based on the clinical laboratory reference ranges of the study center):
- Hematology: Absolute neutrophil count (ANC) ≥1.5×10⁹/L; no abnormalities unrelated to ITP, except a) PLT <20×10⁹/L at Day 1 or within 48 hours of Day 1 is allowed; b) Hemoglobin: If anemia is clearly due to ITP-related bleeding, hemoglobin lower than the lower limit of normal (LLN) may be permitted at investigator's discretion.
- Biochemistry: Total bilirubin (TBIL) ≤1.5×ULN; ALT, AST, ALP ≤3×ULN; serum creatinine ≤1.5×ULN and creatinine clearance ≥50 mL/min.
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- Coagulation: Prothrombin time (PT) within ±3 seconds of normal range; activated partial thromboplastin time (APTT) ≤1.5×ULN unless on medications known to alter INR/APTT; no known history of coagulopathy other than ITP.
- Prior ITP rescue treatments (e.g., platelet transfusion, IVIG, immunomodulators, cyclophosphamide) must have ended or failed at least 2 weeks prior to enrollment (except corticosteroids); TPO-RAs or corticosteroids must be discontinued ≥14 days before study start.
- Patients on maintenance immunosuppressive therapy (e.g., corticosteroids, azathioprine, danazol, cyclosporine A, mycophenolate mofetil, or traditional Chinese platelet-raising medicine) must be on stable doses for at least 1 month prior to enrollment. Patients previously treated with anti-CD20 antibodies must have discontinued at least 6 months prior; splenectomy patients may enroll ≥6 months post-surgery.
- Female subjects of childbearing potential must have a negative serum pregnancy test within 24 hours prior to first dosing; all subjects agree to use effective contraception during the study and for 6 months after its completion.
- No known contraindications to corticosteroids, rhTPO, or eltrombopag and willing to undergo rhTPO followed by eltrombopag treatment.
- Willingness to participate and ability to provide written informed consent. For participants aged ≥12 years, both the subject and their legal guardian must sign the informed consent form.
Who cannot join
- Refractory ITP (failure to respond to first- and second-line TPO-RAs, anti-CD20 therapy, or splenectomy);
- Pregnant or lactating women;
- Evidence of secondary ITP (e.g., untreated Helicobacter pylori, leukemia, lymphoma, autoimmune diseases such as SLE, Hashimoto's thyroiditis), drug-induced thrombocytopenia (e.g., due to anticonvulsants, antibiotics, heparin), or multi-lineage autoimmune cytopenias (e.g., Evans syndrome);
- History or presence of any primary disease other than ITP that may cause thrombocytopenia (e.g., myelodysplastic syndrome, congenital bone marrow failure syndromes such as Fanconi anemia or dyskeratosis congenita, aplastic anemia), as judged by the investigator;
- History of intracranial hemorrhage or severe bleeding (>CTCAE Grade 3) of vital organs; symptomatic gastrointestinal bleeding (e.g., hematemesis, melena) within 6 months prior to screening (asymptomatic occult blood and hemorrhoids excluded);
- History of any arterial or venous thrombosis (e.g., stroke, TIA, MI, DVT, PE) within 6 months prior to enrollment and at least two of the following risk factors: hormone replacement therapy, oral contraceptives (including estrogens), smoking, diabetes, hypercholesterolemia, pharmacologically controlled hypertension, hereditary thrombophilia;
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- Severe cardiovascular disease within 6 months prior to enrollment (e.g., NYHA Class III-IV), arrhythmias increasing thrombotic risk (e.g., atrial fibrillation), or history of coronary stenting, angioplasty, or bypass surgery;
- Concurrent serious or life-threatening malignancy;
- Use of drugs that significantly affect platelet function (e.g., aspirin, clopidogrel, NSAIDs) or anticoagulants for >3 days within 2 weeks before and during the study;
- Use of any herbal medicine or nutritional supplement within 1 week prior to study start, except for vitamins and minerals;
- Ongoing or uncontrolled serious infections (≥CTCAE Grade 2);
- Laboratory or clinical evidence of HIV infection, active hepatitis C, active hepatitis B, or previous history of these infections. Patients with positive HBsAg are excluded. Those with HBsAg-negative but HBcAb-positive must undergo HBV DNA testing; if positive, they are excluded;
- Any condition deemed by the investigator to make the subject unsuitable for participation, including but not limited to medical, social, or psychological factors affecting safety or study compliance.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Wuhan Union Hospital
Wuhan, Hubei, 430022,
Heng Mei, 13886160811, hmei@hust.edu.cn
About this study
Official title: Efficacy and Safety of Glucocorticoids Combined With Recombinant Human Thrombopoietin (rhTPO) Sequential Eltrombopag Olamine for Severe Primary Immune Thrombocytopenia (ITP) in Adults
To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. In this study, hospitalized participants will receive a 14-day regimen of high-dose dexamethasone (HD-DXM) plus recombinant human thrombopoietin (rhTPO), followed by a 10-week course of oral eltrombopag olamine dry suspension after discharge. The investigators aim to evaluate the efficacy and safety of this sequential treatment strategy in adult ITP patients.
Read the detailed description
To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. This approach is based on the superior early response achieved with recombinant human thrombopoietin (rhTPO) combined with high-dose dexamethasone (HD-DXM), and the favorable efficacy of eltrombopag in maintaining long-term responses and reducing relapse. In this study, hospitalized participants will receive a 14-day regimen of HD-DXM plus rhTPO, followed by a 10-week course of oral eltrombopag olamine dry suspension after discharge. The investigators aim to evaluate the efficacy and safety of this sequential treatment strategy in adult ITP participants, with the goal of enhancing early response, sustaining remission, reducing relapse, and minimizing adverse effects. This study is expected to provide evidence-based support for optimizing ITP treatment.
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Eltrombopag, Placebo
- Keywords
- immune thrombocytopenia (ITP), recombinant human thrombopoietin (rhTPO), Eltrombopag
- 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.
EltrombopagDrug
Also called EPAG-pfos
Participants assigned to the observation group will be treated with eltrombopag oral suspension (EPAG-pfos).
PlaceboOther
Participants in the control group will receive a placebo at the same dose, instead of eltrombopag oral suspension (EPAG-pfos).
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag Group | Experimental | Participants who achieve a complete response (CR) or response (R) after HD-DXM combined with rhTPO treatment will be randomized in a 2:1 ratio into the eltrombopag group or the placebo group.
Observation group: Participants will receive eltrombopag for oral suspension (EPAG-pfos) for ten weeks. Receives: Drug: Eltrombopag |
| Placebo group | Active comparator | Participants who achieve a complete response (CR) or response (R) after HD-DXM combined with rhTPO treatment will be randomized in a 2:1 ratio into the eltrombopag group or the placebo group.
Control group: Participants did not receive sequential EPAG-pfos treatment. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sustained response rate (SRR) at 3 months of treatment.
Sustained response rate (SRR) at 3 months of treatment.
Measured: 3 months of the study
Secondary outcomes (7)
The proportions of patients achieving response (R);
The proportions of patients achieving response (R);
Measured: 6 months of the study
Platelet count levels at 3 months after treatment
Platelet count levels at 3 months after treatment
Measured: 3 months of the study
The proportions of patients achieving complete response (CR);
The proportions of patients achieving complete response (CR);
Measured: 6 months of the study
The proportions of patients achieving and platelet counts ≥50×10⁹/L;
The proportions of patients achieving and platelet counts ≥50×10⁹/L;
Measured: 6 months of the study
Sustained response rate (SRR) at 6 months;
Sustained response rate (SRR) at 6 months;
Measured: 6 months of the study
Time to response (TTR);
Time to response (TTR);
Measured: 6 months of the study
Time from treatment initiation to first achievement of PLT ≥100×10⁹/L;
Time from treatment initiation to first achievement of PLT ≥100×10⁹/L;
Measured: 6 months of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2025
- Study start1 Dec 2025 (estimated)
- Primary completion1 Nov 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, MEI HENG, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
- Investigators
- Heng Mei, Principal investigator, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07189910
- Sponsor's protocol ID
- ITP-TPO+Eltro
- Status last verified
- Sep 2025
- First submitted
- 12 Aug 2025
- Last updated
- 24 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: It is not clear whether the data would involve patient privacy or whether patients would agree to the disclosure of the data.