Persistent Isolated Chemotherapy-Induced ThrombocytopeniaPhase 2N01-A-PICIT-GTNCT07586826
Romiplostim N01 Plus ATRA for Persistent Isolated Chemotherapy-Induced Thrombocytopenia After Complete Remission of Gynecologic, Breast, or Lung Solid Tumors
A Phase 2 study testing Romiplostim N01 and All-trans retinoic acid in people with Persistent Isolated Chemotherapy-Induced Thrombocytopenia and Chemotherapy-Induced Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
Trial summary
This is a prospective, randomized, open-label, active-controlled study to evaluate the efficacy and safety of Romiplostim N01 plus all-trans retinoic acid (ATRA) compared with Romiplostim N01 alone in adults with persistent isolated chemotherapy-induced thrombocytopenia (PICIT) after complete remission of selected… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Persistent Isolated Chemotherapy-Induced Thrombocytopenia, Chemotherapy-Induced Thrombocytopenia, Gynecologic Cancers
- What is being tested?
- Romiplostim N01 and All-trans retinoic acid
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 220 participants
- Where
- 1 siteChina
- Study dates
- 22 Dec 2025to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 years or older.
- Prior diagnosis of a selected gynecologic, breast, or lung solid tumor, including but not limited to non-small cell lung cancer (NSCLC), ovarian cancer, or breast cancer. Other eligible tumor types may include endometrial cancer, cervical cancer, fallopian tube cancer, primary peritoneal cancer, and other lung cancers, if clinically appropriate and if all other eligibility criteria are met.
- Complete remission of the underlying tumor after chemotherapy or antitumor treatment, with tumor-related treatment discontinued for at least 12 weeks before enrollment, no evidence of recurrence or progression by specialist assessment, and no current need for additional tumor-directed therapy.
- Persistent isolated chemotherapy-induced thrombocytopenia, defined as platelet count <30 x 10^9/L on two peripheral blood tests at least 7 days apart; or platelet count slightly higher than 30 x 10^9/L with dependence on platelet transfusion to maintain a safe platelet level.
- Thrombocytopenia has persisted since the last chemotherapy treatment without a clear trend of spontaneous recovery.
- Red blood cell count and neutrophil count are generally preserved, without clinically significant anemia or neutropenia.
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- Bone marrow assessment performed within 1 year after tumor diagnosis and chemotherapy shows no tumor cell infiltration; megakaryocyte count is normal or increased, with or without maturation impairment.
- No hepatosplenomegaly, portal hypertension, or other evidence suggesting abnormal platelet redistribution as the main cause of thrombocytopenia.
- Prior treatment with at least one thrombopoietin receptor agonist or recombinant human thrombopoietin for PICIT without response, defined as failure of platelet count to rise to a safe level or to at least 2 times baseline after at least 2 weeks of standard-dose treatment.
- No prior use of Romiplostim N01.
- Other platelet-raising medications have been discontinued before enrollment. No washout period is required for prior thrombopoietin receptor agonists; other investigational drugs or off-label treatments must be discontinued for at least 1 month before enrollment.
- Ability to understand and sign the informed consent form and willingness to comply with study visits and procedures.
- Participants of reproductive potential must agree to use effective contraception during study treatment. Female participants of childbearing potential must have a negative pregnancy test before enrollment.
Who cannot join
- Other hematologic diseases that may affect hematopoiesis or cause thrombocytopenia, including but not limited to aplastic anemia, myelodysplastic syndrome, leukemia or other hematologic malignancies, or a clear history of primary immune thrombocytopenia.
- Active recurrence or progression of the underlying tumor, or evidence of bone marrow metastasis or tumor cell infiltration on bone marrow examination.
- Uncontrolled chronic viral infection, including hepatitis B, hepatitis C, or HIV infection, or active severe infection at screening or within 4 weeks before screening.
- Severe cardiac, hepatic, renal, or other organ dysfunction, or any serious organic disease that would make the participant unable to tolerate study treatment.
- Pregnancy or breastfeeding.
- Known severe hypersensitivity to Romiplostim, Romiplostim N01, ATRA, or any component of the study drugs.
- Prior Romiplostim treatment associated with severe adverse reactions or lack of efficacy.
- Poor compliance, inability to complete treatment or follow-up, psychiatric or psychological condition that prevents understanding of the study procedures, or any other condition that, in the investigator's judgment, may increase study risk or interfere with interpretation of study results.
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, 1 recruiting
- Recruiting
Peking University People's Hospital
Beijing, 100044
Xiaohui Zhang, MD, +8610-8832-4672, zhangxh@bjmu.edu.cn
Kexin Shen, MD, +8617710635036, keceyshen@gmail.com
About this study
Official title: A Prospective, Randomized, Open-Label, Controlled Study of Romiplostim N01 Combined With All-Trans Retinoic Acid Versus Romiplostim N01 Alone for Persistent Isolated Chemotherapy-Induced Thrombocytopenia in Patients With Complete Remission of Gynecologic, Breast, or Lung Solid Tumors
This is a prospective, randomized, open-label, active-controlled study to evaluate the efficacy and safety of Romiplostim N01 plus all-trans retinoic acid (ATRA) compared with Romiplostim N01 alone in adults with persistent isolated chemotherapy-induced thrombocytopenia (PICIT) after complete remission of selected gynecologic, breast, or lung solid tumors, including but not limited to non-small cell lung cancer (NSCLC), ovarian cancer, and breast cancer.
Eligible participants will be randomized in a 1:1 ratio to receive Romiplostim N01 plus oral ATRA or Romiplostim N01 alone for 12 weeks, with follow-up through Week 24. The primary outcome is the overall platelet response rate at Week 12, defined as platelet count >50 x 10^9/L in at least 2 of the last 3 scheduled platelet assessments up to Week 12. Secondary outcomes include sustained response during Weeks 13 to 24, complete and partial response rates, duration of response, time to response, platelet count changes, platelet transfusion requirements, bleeding events, and safety.
Read the detailed description
Chemotherapy-induced thrombocytopenia is a clinically important complication of anticancer treatment. In some patients, thrombocytopenia persists after completion of chemotherapy despite complete remission of the underlying tumor. Persistent isolated chemotherapy-induced thrombocytopenia (PICIT) is characterized by prolonged thrombocytopenia with relatively preserved red blood cell and neutrophil counts, after exclusion of other causes of thrombocytopenia.
This study focuses on adult patients with PICIT after complete remission of selected gynecologic, breast, or lung solid tumors, including but not limited to non-small cell lung cancer (NSCLC), ovarian cancer, and breast cancer. Other eligible tumor types may include endometrial cancer, cervical cancer, fallopian tube cancer, primary peritoneal cancer, and other lung cancers, if the participant otherwise meets the protocol-defined criteria.
Romiplostim N01 is a thrombopoietin receptor agonist intended to stimulate megakaryocyte proliferation and platelet production. All-trans retinoic acid (ATRA) may provide additional hematopoietic and immunomodulatory effects. The study will compare Romiplostim N01 plus ATRA with Romiplostim N01 alone.
Eligible participants will be centrally randomized in a 1:1 ratio to one of two treatment arms. Participants in the experimental arm will receive Romiplostim N01 by subcutaneous injection once weekly plus oral ATRA twice daily for 12 weeks. Participants in the active comparator arm will receive Romiplostim N01 alone once weekly for 12 weeks. Romiplostim N01 dose adjustment will be based on platelet count according to the protocol. Supportive care and rescue treatment, including platelet transfusion for severe bleeding or clinically indicated thrombocytopenia, are permitted.
Participants will be followed weekly during Weeks 1 to 12 and every 2 weeks during Weeks 13 to 24. The primary endpoint is overall response rate at Week 12. Secondary endpoints include sustained response rate during Weeks 13 to 24, complete response rate, partial response rate, duration of response, time to response, platelet count changes, platelet transfusion requirements, bleeding events, and adverse events. Safety will be assessed by monitoring adverse events and serious adverse events, including ATRA-related toxicities and thrombopoietin receptor agonist-associated risks such as thromboembolic events.
- Conditions
- Persistent Isolated Chemotherapy-Induced Thrombocytopenia, Chemotherapy-Induced Thrombocytopenia, Gynecologic Cancers, Ovarian Cancer, Breast Cancer, Lung Cancer
- Being tested
- Romiplostim N01, All-trans retinoic acid
- Keywords
- PICIT, Persistent isolated chemotherapy-induced thrombocytopenia, CIT, Chemotherapy-induced thrombocytopenia, Romiplostim N01, ATRA, All-trans retinoic acid, Tretinoin, Thrombopoietin receptor agonist, Gynecologic cancer, Ovarian cancer, Breast cancer, Lung cancer, NSCLC, Non-small cell lung cancer, Endometrial cancer, Cervical cancer, Complete remission, Platelet response, Thrombocytopenia
- Medical subject headings
- Ovarian Neoplasms, Breast Neoplasms, Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Uterine Cervical Neoplasms, Fallopian Tube Neoplasms, Endometrial Neoplasms, Pathologic Complete Response
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Romiplostim N01Drug
Also called N01, TPO receptor agonist, Thrombopoietin receptor agonist
Romiplostim N01 will be administered by subcutaneous injection at an initial dose of 4 mcg/kg once weekly for 12 weeks. The dose may be adjusted according to platelet count: increase by 2 mcg/kg if platelet count is <50 x 10^9/L, with a maximum dose of 10 mcg/kg; maintain the current dose if platelet count is >=50 to <=200 x 10^9/L; reduce by 1 mcg/kg if platelet count is >200 to <=400 x 10^9/L; and withhold dosing if platelet count is >400 x 10^9/L, then restart at a lower dose after platelet count decreases to approximately 200 x 10^9/L.
All-trans retinoic acidDrug
Also called ATRA, Tretinoin
All-trans retinoic acid (ATRA) will be administered orally at 10 mg twice daily for 12 weeks. Dose interruption, reduction, or discontinuation may be performed for intolerable toxicity or clinically significant adverse events according to the protocol.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim N01 Plus ATRA | Experimental | Participants randomized to this arm will receive Romiplostim N01 by subcutaneous injection once weekly for 12 weeks, with protocol-defined dose adjustment based on platelet count, plus oral all-trans retinoic acid (ATRA) 10 mg twice daily for 12 weeks. Supportive care and rescue treatment, including platelet transfusion when clinically indicated, are permitted according to the protocol. Receives: Drug: Romiplostim N01; Drug: All-trans retinoic acid |
| Romiplostim N01 Alone | Active comparator | Participants randomized to this arm will receive Romiplostim N01 by subcutaneous injection once weekly for 12 weeks, with protocol-defined dose adjustment based on platelet count. Supportive care and rescue treatment, including platelet transfusion when clinically indicated, are permitted according to the protocol. Participants in this arm will not receive ATRA. Receives: Drug: Romiplostim N01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Platelet Response Rate at Week 12
Percentage of participants with platelet count >50 x 10^9/L in at least 2 of the last 3 scheduled platelet assessments up to Week 12. Platelet transfusion or other rescue/supportive treatment will not be counted as a platelet response.
Measured: From randomization to Week 12
Secondary outcomes (10)
Sustained Platelet Response Rate During Weeks 13 to 24
Percentage of participants with platelet count >50 x 10^9/L in at least 2 of the last 3 scheduled platelet assessments during Weeks 13 to 24 and without rescue therapy during this period.
Measured: Weeks 13 through 24
Complete Response Rate
Percentage of participants with platelet count >=100 x 10^9/L and no bleeding for at least 2 consecutive weeks.
Measured: Up to Week 24
Partial Response Rate
Percentage of participants with platelet count >=30 x 10^9/L and at least 2 times the baseline platelet count, with no bleeding.
Measured: Up to Week 24
Duration of Response
Among responders, time from first achievement of platelet response to platelet count <30 x 10^9/L or the end of study follow-up.
Measured: From first documented response to Week 24
Time to Response
Time from treatment start to the first platelet count >=30 x 10^9/L.
Measured: From treatment start to Week 24
Change in Platelet Count Over Time
Change in peripheral blood platelet count from baseline at scheduled study visits.
Measured: Baseline; weekly during Weeks 1 to 12; every 2 weeks during Weeks 13 to 24
Platelet Transfusion Requirement
Frequency and total amount of platelet transfusions during treatment and follow-up.
Measured: From treatment start to Week 24
Incidence of Bleeding Events
Percentage of participants with any bleeding event and severity of bleeding events recorded during the study.
Measured: From treatment start to Week 24
Incidence of Adverse Events and Serious Adverse Events
Adverse events and serious adverse events will be recorded and graded according to CTCAE version 5.0 and summarized by treatment arm.
Measured: From first dose to Week 24
Incidence of Thromboembolic Events
Percentage of participants with confirmed thromboembolic events during treatment and follow-up.
Measured: From first dose to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study start22 Dec 2025
- Primary completion31 Oct 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.
- Publications (12)Papers the sponsor has linked to this study.
- Other trials for Persistent Isolated Chemotherapy-Induced ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Eligible adult participants with persistent isolated chemotherapy-induced thrombocytopenia after complete remission of selected gynecologic, breast, or lung solid tumors will be randomized in a 1:1 ratio to receive either Romiplostim N01 plus ATRA or Romiplostim N01 alone. Treatment will continue for 12 weeks, followed by observation through Week 24. Randomization will be stratified by tumor category, such as NSCLC/lung cancer, ovarian or other gynecologic cancer, and breast cancer, as specified in the statistical analysis plan.
- Masking
- None (open label)
- Enrolment
- 220 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University Cancer Hospital and Institute, Cancer Institute and Hospital, Chinese Academy of Medical Sciences, Peking University International Hospital, Shanxi Province Cancer Hospital, Henan Provincial People's Hospital, China-Japan Union Hospital, Sun Yat-sen University Cancer Center, Hebei Medical University Fourth Hospital, Tianjin Medical University Cancer Institute and Hospital
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
- Investigators
- Xiaohui Zhang, MD, Principal investigator, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07586826
- Sponsor's protocol ID
- 2025PHD035-001-GT
- Acronym
- N01-A-PICIT-GT
- Status last verified
- May 2026
- First submitted
- 8 May 2026
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: The plan for sharing de-identified individual participant data has not yet been finalized. Any future data sharing will require approval by the sponsor and institutional ethics committee, compliance with the informed consent form and applicable privacy regulations, and execution of an appropriate data use agreement. No identifiable participant information will be shared.
Publications
Rainone M, Kasparian S, Nguyen T, Talwar N, Yuan Y, Mei M, Mortimer JE, Waisman JR, Patel N, Pullarkat V. Thrombopoietin Receptor Agonists for Thrombocytopenia Secondary to HER2-Targeted Antibody Drug Conjugates. Oncologist. 2023 Sep 7;28(9):e843-e846. doi: 10.1093/oncolo/oyad185.
PubMed 37335880Schweinfurth N, Hohmann S, Deuschle M, Lederbogen F, Schloss P. Valproic acid and all trans retinoic acid differentially induce megakaryopoiesis and platelet-like particle formation from the megakaryoblastic cell line MEG-01. Platelets. 2010;21(8):648-57. doi: 10.3109/09537104.2010.513748. Epub 2010 Oct 13.
PubMed 20942599Zhu X, Wang Y, Jiang Q, Jiang H, Lu J, Wang Y, Kong Y, Chang Y, Xu L, Peng J, Hou M, Huang X, Zhang X. All-trans retinoic acid protects mesenchymal stem cells from immune thrombocytopenia by regulating the complement-interleukin-1beta loop. Haematologica. 2019 Aug;104(8):1661-1675. doi: 10.3324/haematol.2018.204446. Epub 2019 Jan 24.
PubMed 30679324Wu YH, Chen HY, Hong WC, Wei CY, Pang JS. Carboplatin-Induced Thrombocytopenia through JAK2 Downregulation, S-Phase Cell Cycle Arrest and Apoptosis in Megakaryocytes. Int J Mol Sci. 2022 Jun 3;23(11):6290. doi: 10.3390/ijms23116290.
PubMed 35682967Al-Samkari H, Kolb-Sielecki J, Safina SZ, Xue X, Jamieson BD. Avatrombopag for chemotherapy-induced thrombocytopenia in patients with non-haematological malignancies: an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2022 Mar;9(3):e179-e189. doi: 10.1016/S2352-3026(22)00001-1.
PubMed 35240074Wilkins CR, Ortiz J, Gilbert LJ, Yin S, Mones JV, Parameswaran R, Mantha S, Soff GA. Romiplostim for chemotherapy-induced thrombocytopenia: Efficacy and safety of extended use. Res Pract Thromb Haemost. 2022 May 10;6(3):e12701. doi: 10.1002/rth2.12701. eCollection 2022 Mar.
PubMed 35582038Soff GA, Miao Y, Bendheim G, Batista J, Mones JV, Parameswaran R, Wilkins CR, Devlin SM, Abou-Alfa GK, Cercek A, Kemeny NE, Sarasohn DM, Mantha S. Romiplostim Treatment of Chemotherapy-Induced Thrombocytopenia. J Clin Oncol. 2019 Nov 1;37(31):2892-2898. doi: 10.1200/JCO.18.01931. Epub 2019 Sep 23.
PubMed 31545663Moufarrij S, O'Cearbhaill RE, Zhou Q, Iasonos A, Mantha S, Zwicker J, Wilkins CR. Use of romiplostim for antineoplastic therapy-induced thrombocytopenia in gynecologic and breast cancers. Gynecol Oncol Rep. 2024 Apr 24;53:101399. doi: 10.1016/j.gore.2024.101399. eCollection 2024 Jun.
PubMed 38757118Xu Y, Song X, Du F, Zhao Q, Liu L, Ma Z, Lu S. A Randomized Controlled Study of rhTPO and rhIL-11 for the Prophylactic Treatment of Chemotherapy-Induced Thrombocytopenia in Non-Small Cell Lung Cancer. J Cancer. 2018 Nov 25;9(24):4718-4725. doi: 10.7150/jca.26690. eCollection 2018.
PubMed 30588257Goldberg GL, Gibbon DG, Smith HO, DeVictoria C, Runowicz CD, Burns ER. Clinical impact of chemotherapy-induced thrombocytopenia in patients with gynecologic cancer. J Clin Oncol. 1994 Nov;12(11):2317-20. doi: 10.1200/JCO.1994.12.11.2317.
PubMed 7964946Adelborg K, Veres K, Horvath-Puho E, Clouser M, Saad H, Sorensen HT. Risk and adverse clinical outcomes of thrombocytopenia among patients with solid tumors-a Danish population-based cohort study. Br J Cancer. 2024 May;130(9):1485-1492. doi: 10.1038/s41416-024-02630-w. Epub 2024 Mar 6.
PubMed 38448749Shaw JL, Nielson CM, Park JK, Marongiu A, Soff GA. The incidence of thrombocytopenia in adult patients receiving chemotherapy for solid tumors or hematologic malignancies. Eur J Haematol. 2021 May;106(5):662-672. doi: 10.1111/ejh.13595. Epub 2021 Feb 16.
PubMed 33544940
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Randomized, Open-Label, Controlled Study of Romiplostim N01 Combined With All-Trans Retinoic Acid Versus Romiplostim N01 Alone for Persistent Isolated Chemotherapy-Induced Thrombocytopenia in Patients With Complete Remission of Gynecologic, Breast, or Lung Solid Tumors
- ClinicalTrials.gov ID
- NCT07586826
- Acronym / sponsor ID
- N01-A-PICIT-GT / 2025PHD035-001-GT
- Conditions
- Persistent Isolated Chemotherapy-Induced Thrombocytopenia, Chemotherapy-Induced Thrombocytopenia, Gynecologic Cancers, Ovarian Cancer, Breast Cancer, Lung Cancer
- Keywords
- PICIT, Persistent isolated chemotherapy-induced thrombocytopenia, CIT, Chemotherapy-induced thrombocytopenia, Romiplostim N01, ATRA, All-trans retinoic acid, Tretinoin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 220 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Peking University Cancer Hospital and Institute, Cancer Institute and Hospital, Chinese Academy of Medical Sciences, Peking University International Hospital, Shanxi Province Cancer Hospital, Henan Provincial People's Hospital, China-Japan Union Hospital, Sun Yat-sen University Cancer Center, Hebei Medical University Fourth Hospital, Tianjin Medical University Cancer Institute and Hospital
- Study start
- 22 Dec 2025 (actual)
- Primary completion
- 31 Oct 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 14 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective, randomized, open-label, active-controlled study to evaluate the efficacy and safety of Romiplostim N01 plus all-trans retinoic acid (ATRA) compared with Romiplostim N01 alone in adults with persistent isolated chemotherapy-induced thrombocytopenia (PICIT) after complete remission of selected gynecologic, breast, or lung solid tumors, including but not limited to non-small cell lung cancer (NSCLC), ovarian cancer, and breast cancer.
Eligible participants will be randomized in a 1:1 ratio to receive Romiplostim N01 plus oral ATRA or Romiplostim N01 alone for 12 weeks, with follow-up through Week 24. The primary outcome is the overall platelet response rate at Week 12, defined as platelet count >50 x 10^9/L in at least 2 of the last 3 scheduled platelet assessments up to Week 12. Secondary outcomes include sustained response during Weeks 13 to 24, complete and partial response rates, duration of response, time to response, platelet count changes, platelet transfusion requirements, bleeding events, and safety.
Detailed description
Chemotherapy-induced thrombocytopenia is a clinically important complication of anticancer treatment. In some patients, thrombocytopenia persists after completion of chemotherapy despite complete remission of the underlying tumor. Persistent isolated chemotherapy-induced thrombocytopenia (PICIT) is characterized by prolonged thrombocytopenia with relatively preserved red blood cell and neutrophil counts, after exclusion of other causes of thrombocytopenia.
This study focuses on adult patients with PICIT after complete remission of selected gynecologic, breast, or lung solid tumors, including but not limited to non-small cell lung cancer (NSCLC), ovarian cancer, and breast cancer. Other eligible tumor types may include endometrial cancer, cervical cancer, fallopian tube cancer, primary peritoneal cancer, and other lung cancers, if the participant otherwise meets the protocol-defined criteria.
Romiplostim N01 is a thrombopoietin receptor agonist intended to stimulate megakaryocyte proliferation and platelet production. All-trans retinoic acid (ATRA) may provide additional hematopoietic and immunomodulatory effects. The study will compare Romiplostim N01 plus ATRA with Romiplostim N01 alone.
Eligible participants will be centrally randomized in a 1:1 ratio to one of two treatment arms. Participants in the experimental arm will receive Romiplostim N01 by subcutaneous injection once weekly plus oral ATRA twice daily for 12 weeks. Participants in the active comparator arm will receive Romiplostim N01 alone once weekly for 12 weeks. Romiplostim N01 dose adjustment will be based on platelet count according to the protocol. Supportive care and rescue treatment, including platelet transfusion for severe bleeding or clinically indicated thrombocytopenia, are permitted.
Participants will be followed weekly during Weeks 1 to 12 and every 2 weeks during Weeks 13 to 24. The primary endpoint is overall response rate at Week 12. Secondary endpoints include sustained response rate during Weeks 13 to 24, complete response rate, partial response rate, duration of response, time to response, platelet count changes, platelet transfusion requirements, bleeding events, and adverse events. Safety will be assessed by monitoring adverse events and serious adverse events, including ATRA-related toxicities and thrombopoietin receptor agonist-associated risks such as thromboembolic events.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Eligible adult participants with persistent isolated chemotherapy-induced thrombocytopenia after complete remission of selected gynecologic, breast, or lung solid tumors will be randomized in a 1:1 ratio to receive either Romiplostim N01 plus ATRA or Romiplostim N01 alone. Treatment will continue for 12 weeks, followed by observation through Week 24. Randomization will be stratified by tumor category, such as NSCLC/lung cancer, ovarian or other gynecologic cancer, and breast cancer, as specified in the statistical analysis plan.
| Arm | Type | Receives |
|---|---|---|
| Romiplostim N01 Plus ATRA | Experimental | Drug: Romiplostim N01, Drug: All-trans retinoic acidParticipants randomized to this arm will receive Romiplostim N01 by subcutaneous injection once weekly for 12 weeks, with protocol-defined dose adjustment based on platelet count, plus oral all-trans retinoic acid (ATRA) 10 mg twice daily for 12 weeks. Supportive care and rescue treatment, including platelet transfusion when clinically indicated, are permitted according to the protocol. |
| Romiplostim N01 Alone | Active comparator | Drug: Romiplostim N01Participants randomized to this arm will receive Romiplostim N01 by subcutaneous injection once weekly for 12 weeks, with protocol-defined dose adjustment based on platelet count. Supportive care and rescue treatment, including platelet transfusion when clinically indicated, are permitted according to the protocol. Participants in this arm will not receive ATRA. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Age 18 years or older.
- Prior diagnosis of a selected gynecologic, breast, or lung solid tumor, including but not limited to non-small cell lung cancer (NSCLC), ovarian cancer, or breast cancer. Other eligible tumor types may include endometrial cancer, cervical cancer, fallopian tube cancer, primary peritoneal cancer, and other lung cancers, if clinically appropriate and if all other eligibility criteria are met.
- Complete remission of the underlying tumor after chemotherapy or antitumor treatment, with tumor-related treatment discontinued for at least 12 weeks before enrollment, no evidence of recurrence or progression by specialist assessment, and no current need for additional tumor-directed therapy.
- Persistent isolated chemotherapy-induced thrombocytopenia, defined as platelet count <30 x 10^9/L on two peripheral blood tests at least 7 days apart; or platelet count slightly higher than 30 x 10^9/L with dependence on platelet transfusion to maintain a safe platelet level.
- Thrombocytopenia has persisted since the last chemotherapy treatment without a clear trend of spontaneous recovery.
Key exclusion criteria (8 listed)
- Other hematologic diseases that may affect hematopoiesis or cause thrombocytopenia, including but not limited to aplastic anemia, myelodysplastic syndrome, leukemia or other hematologic malignancies, or a clear history of primary immune thrombocytopenia.
- Active recurrence or progression of the underlying tumor, or evidence of bone marrow metastasis or tumor cell infiltration on bone marrow examination.
- Uncontrolled chronic viral infection, including hepatitis B, hepatitis C, or HIV infection, or active severe infection at screening or within 4 weeks before screening.
- Severe cardiac, hepatic, renal, or other organ dysfunction, or any serious organic disease that would make the participant unable to tolerate study treatment.
- Pregnancy or breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Romiplostim N01Also: N01, TPO receptor agonist, Thrombopoietin receptor agonistRomiplostim N01 will be administered by subcutaneous injection at an initial dose of 4 mcg/kg once weekly for 12 weeks. The dose may be adjusted according to platelet count: increase by 2 mcg/kg if platelet count is <50 x 10^9/L, with a maximum dose of 10 mcg/kg; maintain the current dose if platelet count is >=50 to <=200 x 10^9/L; reduce by 1 mcg/kg if platelet count is >200 to <=400 x 10^9/L; and withhold dosing if platelet count is >400 x 10^9/L, then restart at a lower dose after platelet count decreases to approximately 200 x 10^9/L. | Drug | Romiplostim N01 Alone, Romiplostim N01 Plus ATRA |
| All-trans retinoic acidAlso: ATRA, TretinoinAll-trans retinoic acid (ATRA) will be administered orally at 10 mg twice daily for 12 weeks. Dose interruption, reduction, or discontinuation may be performed for intolerable toxicity or clinically significant adverse events according to the protocol. | Drug | Romiplostim N01 Plus ATRA |
Primary outcome
Overall Platelet Response Rate at Week 12
Time frame: From randomization to Week 12
Percentage of participants with platelet count >50 x 10^9/L in at least 2 of the last 3 scheduled platelet assessments up to Week 12. Platelet transfusion or other rescue/supportive treatment will not be counted as a platelet response.
10 secondary outcomes
Sustained Platelet Response Rate During Weeks 13 to 24
Time frame: Weeks 13 through 24
Complete Response Rate
Time frame: Up to Week 24
Partial Response Rate
Time frame: Up to Week 24
Duration of Response
Time frame: From first documented response to Week 24
Time to Response
Time frame: From treatment start to Week 24
Change in Platelet Count Over Time
Time frame: Baseline; weekly during Weeks 1 to 12; every 2 weeks during Weeks 13 to 24
Platelet Transfusion Requirement
Time frame: From treatment start to Week 24
Incidence of Bleeding Events
Time frame: From treatment start to Week 24
Incidence of Adverse Events and Serious Adverse Events
Time frame: From first dose to Week 24
Incidence of Thromboembolic Events
Time frame: From first dose to Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
- Background: PubMed 37335880Rainone M, Kasparian S, Nguyen T, Talwar N, Yuan Y, Mei M, Mortimer JE, Waisman JR, Patel N, Pullarkat V. Thrombopoietin Receptor Agonists for Thrombocytopenia Secondary to HER2-Targeted Antibody Drug Conjugates. Oncologist. 2023 Sep 7;28(9):e843-e846. doi: 10.1093/oncolo/oyad185.
- Background: PubMed 20942599Schweinfurth N, Hohmann S, Deuschle M, Lederbogen F, Schloss P. Valproic acid and all trans retinoic acid differentially induce megakaryopoiesis and platelet-like particle formation from the megakaryoblastic cell line MEG-01. Platelets. 2010;21(8):648-57. doi: 10.3109/09537104.2010.513748. Epub 2010 Oct 13.
- Background: PubMed 30679324Zhu X, Wang Y, Jiang Q, Jiang H, Lu J, Wang Y, Kong Y, Chang Y, Xu L, Peng J, Hou M, Huang X, Zhang X. All-trans retinoic acid protects mesenchymal stem cells from immune thrombocytopenia by regulating the complement-interleukin-1beta loop. Haematologica. 2019 Aug;104(8):1661-1675. doi: 10.3324/haematol.2018.204446. Epub 2019 Jan 24.
- Background: PubMed 35682967Wu YH, Chen HY, Hong WC, Wei CY, Pang JS. Carboplatin-Induced Thrombocytopenia through JAK2 Downregulation, S-Phase Cell Cycle Arrest and Apoptosis in Megakaryocytes. Int J Mol Sci. 2022 Jun 3;23(11):6290. doi: 10.3390/ijms23116290.
- Background: PubMed 35240074Al-Samkari H, Kolb-Sielecki J, Safina SZ, Xue X, Jamieson BD. Avatrombopag for chemotherapy-induced thrombocytopenia in patients with non-haematological malignancies: an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2022 Mar;9(3):e179-e189. doi: 10.1016/S2352-3026(22)00001-1.
- Background: PubMed 35582038Wilkins CR, Ortiz J, Gilbert LJ, Yin S, Mones JV, Parameswaran R, Mantha S, Soff GA. Romiplostim for chemotherapy-induced thrombocytopenia: Efficacy and safety of extended use. Res Pract Thromb Haemost. 2022 May 10;6(3):e12701. doi: 10.1002/rth2.12701. eCollection 2022 Mar.
- Background: PubMed 31545663Soff GA, Miao Y, Bendheim G, Batista J, Mones JV, Parameswaran R, Wilkins CR, Devlin SM, Abou-Alfa GK, Cercek A, Kemeny NE, Sarasohn DM, Mantha S. Romiplostim Treatment of Chemotherapy-Induced Thrombocytopenia. J Clin Oncol. 2019 Nov 1;37(31):2892-2898. doi: 10.1200/JCO.18.01931. Epub 2019 Sep 23.
- Background: PubMed 38757118Moufarrij S, O'Cearbhaill RE, Zhou Q, Iasonos A, Mantha S, Zwicker J, Wilkins CR. Use of romiplostim for antineoplastic therapy-induced thrombocytopenia in gynecologic and breast cancers. Gynecol Oncol Rep. 2024 Apr 24;53:101399. doi: 10.1016/j.gore.2024.101399. eCollection 2024 Jun.
- Background: PubMed 30588257Xu Y, Song X, Du F, Zhao Q, Liu L, Ma Z, Lu S. A Randomized Controlled Study of rhTPO and rhIL-11 for the Prophylactic Treatment of Chemotherapy-Induced Thrombocytopenia in Non-Small Cell Lung Cancer. J Cancer. 2018 Nov 25;9(24):4718-4725. doi: 10.7150/jca.26690. eCollection 2018.
- Background: PubMed 7964946Goldberg GL, Gibbon DG, Smith HO, DeVictoria C, Runowicz CD, Burns ER. Clinical impact of chemotherapy-induced thrombocytopenia in patients with gynecologic cancer. J Clin Oncol. 1994 Nov;12(11):2317-20. doi: 10.1200/JCO.1994.12.11.2317.
- Background: PubMed 38448749Adelborg K, Veres K, Horvath-Puho E, Clouser M, Saad H, Sorensen HT. Risk and adverse clinical outcomes of thrombocytopenia among patients with solid tumors-a Danish population-based cohort study. Br J Cancer. 2024 May;130(9):1485-1492. doi: 10.1038/s41416-024-02630-w. Epub 2024 Mar 6.
- Background: PubMed 33544940Shaw JL, Nielson CM, Park JK, Marongiu A, Soff GA. The incidence of thrombocytopenia in adult patients receiving chemotherapy for solid tumors or hematologic malignancies. Eur J Haematol. 2021 May;106(5):662-672. doi: 10.1111/ejh.13595. Epub 2021 Feb 16.
- Individual participant dataSharing planned: Undecided. The plan for sharing de-identified individual participant data has not yet been finalized. Any future data sharing will require approval by the sponsor and institutional ethics committee, compliance with the informed consent form and applicable privacy regulations, and execution of an appropriate data use agreement. No identifiable participant information will be shared.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Xiaohui Zhang, MD principal investigator
Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Romiplostim N01 and All-trans retinoic acid in people with Persistent Isolated Chemotherapy-Induced Thrombocytopenia and Chemotherapy-Induced Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaohui Zhang, MD (+8610-8832-4672, zhangxh@bjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Platelet Response Rate at Week 12 (time frame: From randomization to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07586826, which also lists 12 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2026; AzTrials last refreshed its copy on 7 Nov 2026.