Romiplostim N01 to treat chemotherapy-related low platelets

Romiplostim N01 for Chemotherapy-induced Thrombocytopenia in Tumors: a Prospective Multi-center One-arm Study

This will test whether Romiplostim N01 can raise platelet counts in adults whose platelets remain very low after chemotherapy and did not respond to other platelet-boosting drugs.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT07063225 on ClinicalTrials.gov

What this trial studies

This is a single-arm Phase 2 trial of Romiplostim N01 in adults with chemotherapy-induced thrombocytopenia who remained refractory after repeated treatment with recombinant human thrombopoietin (rhTPO) or interleukin-11 (IL-11). Eligible patients have platelet counts below 30×10^9/L for more than one month and have stopped chemo/radiotherapy for at least one month. Investigators will give Romiplostim N01 and monitor platelet counts from week 1 through week 24, with weekly safety visits during a 4-week safety period and ongoing adverse event surveillance. Outcomes will be compared against contemporaneous or historical patients receiving supportive care or eltrombopag.

Who should consider this trial

Good fit: Adults (≥18 years) with tumor-related chemotherapy-induced thrombocytopenia with platelets <30×10^9/L for >1 month who failed prior rhTPO or IL-11, have stopped chemo/radiotherapy ≥1 month ago, expected survival ≥6 months, and acceptable liver/kidney and cardiac function are ideal candidates.

Not a fit: Patients whose platelet counts respond to standard supportive agents or who have platelet counts above 30×10^9/L, or those with life expectancy under six months or unacceptable organ dysfunction, are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If effective, Romiplostim N01 could raise platelet counts, lower bleeding risk, and reduce the need for platelet transfusions in patients with refractory chemotherapy-induced thrombocytopenia.

How similar studies have performed: Other thrombopoietin receptor agonists such as eltrombopag and romiplostim have shown the ability to raise platelet counts in similar settings, so the approach has supporting evidence though this specific formulation is less tested.

Eligibility criteria

Inclusion Criteria:

* Age: 18 years or above, gender not restricted;
* Patients who meet the diagnostic criteria for chemotherapy-related thrombocytopenia, and still ineffective after repeated treatment with rhTPO or IL-11 (subjects need to undergo regular rhTPO treatment for at least 14 days after radiotherapy and chemotherapy, with a dose of rhTPO 300u/kg/d or higher, and the platelet count remains less than 30×10\^9/L, defined as refractory CTIT);
* Patients who have stopped chemotherapy and radiotherapy for more than 1 month;
* Patients with platelet count at enrollment \< 30×10\^9/L;
* Patients with platelet count remains \< 30×10\^9/L for more than 1 month;
* Patients with expected survival period ≥ 6 months;
* Patients with liver and kidney function \< 1.5× upper limit of normal value, physical examination qualified;
* Patients with ECOG physical status score ≤ 2 points;
* Patients with Cardiac function: New York Heart Association cardiac function classification is grade 2 or below;
* Patients who is voluntary to sign the informed consent form;

Exclusion Criteria:

* Those with uncontrollable primary diseases of important organs, such as extensive metastasis of malignant tumors, liver failure, heart failure, kidney failure and other diseases；
* Patients with poor compliance;
* Positive serology for HIV, hepatitis B virus (HBV), hepatitis C virus (HCV), and/or hepatitis D virus (HDV), Syphilis; Positive for Epstein-Barr Virus DNA, Cytomegalovirus DNA;
* Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal bleeding, intracranial hemorrhage, etc.
* There is currently a heart disease requiring treatment or a poorly controlled hypertension judged by the investigator；
* Patients with thrombotic diseases such as pulmonary embolism, thrombosis, and atherosclerosis；
* Those who have received allogeneic stem cell transplantation or organ transplantation in the past；
* Patients with mental disorders who cannot normally obtain informed consent and undergo trials and follow-up；
* Patients whose toxic symptoms caused by treatment before participating in the trial have not disappeared；
* Other serious diseases that may restrict participants from participating in this trial (such as diabetes; severe heart failure; myocardial obstruction or unstable arrhythmia or unstable angina in the past 6 months; gastric ulcers; mobility Autoimmune diseases, etc.)；
* Patients with sepsis or patients with other irregular bleeding；
* Patients taking antiplatelet drugs at the same time；
* Pregnant women, suspected pregnancy (a positive pregnancy test for human chorionic gonadotropin in urine at screening) and breastfeeding patients； Pre-existing cardiac disease, including congestive heart failure of New York Heart Association \[NYHA\] Grade III/IV, arrhythmia requiring treatment or myocardial infarction within the last 6 months. No arrhythmia known to increase the risk of thrombotic events (e.g. atrial fibrillation), or patients with a QT \>450msec or QTc \> 480 for patients with a Bundle Branch Block;
* Researchers believe that patients should not participate in the test of any other condition.

Where this trial is running

Tianjin, Tianjin Municipality

• Chinese Academy of Medical Science and Blood Disease Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Principal investigator: Yunfei Chen, MD — Chinese Academy of Medical Science and Blood Disease Hospital
• Study coordinator: Ting Sun, MD

Conditions Chemotherapy-induced Thrombocytopenia; Romiplostim N01