rhTPO Dose Escalation vs Eltrombopag Switch in ITP

NCT07476846 | Not Yet Recruiting

• 1 other identifier: IIT2026001
• Study Type: interventional
• Target: 112
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study is a prospective, multicenter, randomized controlled study, planning to enroll 110 ITP patients who failed to respond to conventional-dose rhTPO (300 IU/kg/d) after 14 days of treatment (PLT \< 30×10⁹/L). After a 2-week washout period, they will be randomized to the rhTPO double-dose group (Group A) and EPAG-pfos group (Group B), with blood routine monitored weekly and doses adjusted according to platelet levels, comparing the response rates of the two groups at 6 weeks after switching treatment.

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

ITP; recombinant human thrombopoietin; eltrombopag

Outcome Measures

Primary Outcomes (1)

• Platelet Response Rate (RR) at 6 weeks after switching treatment: proportion of patients with PLT ≥ 30×10⁹/L, at least 2-fold increase from baseline platelet count, and no bleeding manifestations

  This primary endpoint was defined as the proportion of patients who met the "platelet response" criteria at the 6th week (±1 days) after switching from baseline treatment to the intervention plan of this study. "Platelet response" requires the simultaneous satisfaction of the following three conditions: 1) Platelet count ≥ 30 × 10⁹/L; 2) The platelet count has increased by at least twice compared to the baseline value; 3) There were no bleeding events requiring medical intervention or having clinical significance (WHO bleeding grade 0-1). The calculation formula is: (Number of patients achieving response/total number of patients conforming to the protocol analysis set) × 100%.

  6th week after treatment conversion (day 42, visitation window allowed: Day 41 to day 43)

Secondary Outcomes (5)

• Proportion of patients with at least one PLT ≥ 50×10⁹/L at week 6

  Week 6 after treatment conversion (Day 42, visitation window allowed: Day 41 to day 43)

• Time to Response (TTR)

  The time from the initiation of treatment switching to the first PLT count ≥30×10⁹/L (in days) was recorded. Visiting window allowed: Days 1 to 42.

• Sustained Response Rate (SRR)

  At the 6th or 12th week after treatment conversion (day 42 or day 85 , visitation window allowed: days 41 to 43 or days 82 to 88 ).

• Response Rate (RR), proportion with PLT ≥ 50×10⁹/L, and Complete Response rate (CR, i.e., PLT ≥ 100×10⁹/L without bleeding manifestations) at each visit.

  Weeks 1, 2, 3, 4, 5, 6, 8, 10, and 12 after treatment conversion (according to the protocol visit plan).

• Duration of response

  From the date of first achieving remission (CR/R) until the end of the study visit (week 12，Day 85 ±3) or the date of early termination of the study.

Other Outcomes (6)

• The incidence of serious adverse events (SAE) during treatment

  From the first administration to 14 days (approximately 2 weeks) after the last administration.

• Bleeding events

  From the first study medication to the end of the last follow-up (maximum observation period: 12 weeks).

• Platelet transfusion rate

  From the first study medication to the end of the last follow-up (maximum observation period: 12 weeks).

Study Arms (2)

rhTPO Double-Dose Group (Group A)

EXPERIMENTAL

rhTPO (Shenyang Sunshine Pharmaceutical Co., Ltd., National Medical Product Approval No. S20050048, specification 15000U/ml), starting dose 600 IU/kg/d, continuous subcutaneous injection, blood routine monitored weekly, dose adjusted according to platelet levels. ① Patients with PLT ≥ 50×10⁹/L for two consecutive tests enter maintenance therapy, reducing frequency to alternate-day dosing to maintain PLT at 50-150×10⁹/L; ② When 250×10⁹/L ≥ PLT \> 150×10⁹/L, reduce rhTPO dose to 300 IU/kg/d, alternate-day dosing; ③ When PLT \> 250×10⁹/L, suspend rhTPO. If PLT drops to \<100×10⁹/L, patient may restart rhTPO

Drug: Recombinant Human Thrombopoietin（rhTPO）

EPAG-pfos Group (Group B)

ACTIVE COMPARATOR

EPAG-pfos (Shenyang Sunshine Pharmaceutical Co., Ltd.; specification: 25 mg \[calculated as C25H22N4O4\]) * Administration Separation: Antacids, dairy products, and cationic mineral supplements should be taken at least 2 hours before or at least 4 hours after administration of this product. * Starting Dose: 50 mg daily. * Dose Adjustment: Dose adjusted every 2 weeks based on PLT levels in 25 mg increments (not exceeding 75 mg/d) or dosing frequency: ① If no response at 2 weeks, increase from 50 mg/d to maximum dose 75 mg/d; if ineffective after 2-4 weeks at maximum dose, discontinue; ② When 150×10⁹/L ≥ PLT ≥ 50×10⁹/L, maintain current dose, continuous or alternate-day maintenance therapy; ③ When 250×10⁹/L ≥ PLT \> 150×10⁹/L, reduce dose by 25 mg from current dose, continuous or alternate-day therapy; ④ When PLT \> 250×10⁹/L, suspend EPAG-pfos. If PLT drops to \<100×10⁹/L, patient may restart EPAG-pfos treatment.

Drug: Eltrombopag PfOS

Interventions

Recombinant Human Thrombopoietin（rhTPO） DRUG

rhTPO (Shenyang Sunshine Pharmaceutical Co., Ltd., National Medical Product Approval No. S20050048, specification 15000U/ml), starting dose 600 IU/kg/d, continuous subcutaneous injection, blood routine monitored weekly, dose adjusted according to platelet levels.

rhTPO Double-Dose Group (Group A)

Eltrombopag PfOS DRUG

EPAG-PFOS (Shenyang Sunshine Pharmaceutical Co., Ltd.; 25 mg, calculated as C₂₅H₂₂N₄O₄) * Drug-Food Interactions: Administer at least 2 hours before or 4 hours after antacids, dairy products, or cationic mineral supplements. * Dosing: Initiate at 50 mg once daily; adjust the dose in 25 mg increments (not to exceed 75 mg/day) or modify dosing frequency every 2 weeks based on platelet count.

EPAG-pfos Group (Group B)

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 12-75 years, either sex;
• ECOG performance status 0-1;
• Diagnosis of ITP confirmed by bone marrow biopsy (valid within 3 months) or other relevant examinations;
• Patients who failed short-term rhTPO second-line treatment (≤14 days of medication) (PLT \< 30×10⁹/L);
• Major organ function must meet the following requirements (based on normal values at the clinical trial center):
• Blood routine: absolute neutrophil count (ANC) ≥ 1.5×10⁹/L; no abnormalities other than ITP, except: a) PLT \< 30×10⁹/L at Day 1 visit or within 48 hours of Day 1 is acceptable for enrollment; b) Hemoglobin: if anemia is clearly due to ITP (excessive bleeding related to thrombocytopenia), subjects with hemoglobin below the lower limit of normal may be enrolled based on investigator judgment;
• Blood biochemistry: total bilirubin (TBIL) ≤ 1.5×ULN; ALT, AST, or ALP ≤ 3×ULN; serum creatinine (Cr) ≤ 1.5×ULN with creatinine clearance ≥ 50 mL/min;
• Coagulation function: prothrombin time (PT) within ±3s of normal range; activated partial thromboplastin time (APTT) ≤ 1.5×ULN unless on medications known to alter INR and APTT; no history of coagulation abnormalities other than ITP;
• Previous ITP combination treatments including platelet transfusion, immunoglobulin, immunomodulators, and cyclophosphamide rescue therapy must have ended ≥2 weeks before enrollment; corticosteroids or TPO-class drug treatments must have ended ≥2 weeks before study start;
• Patients on immunosuppressants (including corticosteroids, azathioprine, danazol, cyclosporin A, mycophenolate mofetil) or platelet-elevating traditional Chinese medicine maintenance therapy must have stable therapeutic doses for at least the most recent month; patients receiving CD20 monoclonal antibody must have stopped treatment ≥6 months before enrollment; splenectomy patients may enroll ≥6 months after surgery;
• Women of childbearing potential must have negative serum pregnancy test within 24 hours before first dose; all subjects must agree to use effective contraception during the study and for 6 months after study treatment completion;
• No contraindications to rhTPO and eltrombopag use;
• Voluntary participation in this study, signed informed consent, good compliance, and willingness to cooperate with follow-up.

You may not qualify if:

• Refractory ITP patients (failure of first-line and second-line thrombopoietic drugs and CD20 monoclonal antibody treatment, or splenectomy failure/postoperative relapse);
• Pregnant or lactating patients;
• Evidence of secondary causes of ITP (e.g., untreated Helicobacter pylori infection, leukemia, lymphoma, autoimmune diseases such as SLE, Hashimoto's thyroiditis) or drug-induced (e.g., anticonvulsants, antibiotics, heparin), or bicytopenia/pancytopenia such as Evans syndrome, immune-related cytopenias, etc.;
• History or current presence of primary diseases other than ITP causing thrombocytopenia (e.g., primary myelodysplastic syndrome \[MDS\], congenital bone marrow failure diseases \[e.g., Fanconi anemia, dyskeratosis congenita\], aplastic anemia \[AA\]), and judged by investigator as unsuitable for this study;
• History of intracranial hemorrhage or other important organ severe bleeding (\>CTC AE Grade 3), or history of symptomatic gastrointestinal bleeding (e.g., hematemesis, melena) within 6 months before screening (occult blood test positivity without symptoms/signs and hemorrhoids excluded);
• History of any arterial or venous thrombosis within 6 months before enrollment (including stroke, TIA, MI, DVT, or PE) AND presence of at least 2 of the following risk factors: hormone replacement therapy, oral contraceptives (including estrogen), smoking, diabetes, hypercholesterolemia, drug-controlled hypertension, hereditary coagulation disorders;
• Severe cardiovascular disease within 6 months before enrollment (NYHA Class III-IV), known arrhythmia increasing thromboembolic risk such as atrial fibrillation, coronary stent implantation, angioplasty, or post-CABG patients;
• Coexisting malignancy severely affecting survival;
• Continuous use of medications affecting platelet function (including but not limited to aspirin, clopidogrel, and/or NSAIDs) or anticoagulant therapy \>3 days from 2 weeks after study start until study end;
• Use of any herbal medicine or nutritional supplements within 1 week before study start, except vitamin and mineral supplements;
• Currently having severe or uncontrolled infection (CTC AE Grade 2 infection);
• Laboratory or clinical evidence of HIV infection, previous hepatitis C clinical history, previous hepatitis B infection, or active hepatitis/active tuberculosis at screening. Screening laboratory tests indicating hepatitis C or hepatitis B infection (defined as positive HBsAg; additionally, if HBsAg negative but HBcAb positive, regardless of HBsAb status, HBV DNA testing is required, and if positive, subject should be excluded);
• Patients considered by investigator as unsuitable for this trial due to any other medical, social, or psychological factors that may affect safety or compliance with study procedures.

Sponsors & Collaborators

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

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

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

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2026
• First Posted: March 17, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): March 1, 2028
• Last Updated: July 1, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share