Romiplostim N01 Plus ATRA for Persistent Isolated Chemotherapy-Induced Thrombocytopenia After Complete Remission of Gastrointestinal Solid Tumors

NCT07586813 | Recruiting Phase 2

• 1 other identifier: 2025PHD035-001-GI
• Study Type: interventional
• Target: 220
• Locations: 1 country
• Sites: 1

Brief 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 gastrointestinal/digestive system solid tumors, including but not limited to gastrointestinal tract, pancreatic, and colorectal cancers. 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.

Conditions

Chemotherapy-Induced Thrombocytopenia; Gastrointestinal Neoplasms; Pancreatic Cancer; Colorectal Cancer; Persistent Isolated Chemotherapy-Induced Thrombocytopenia

Keywords

PICIT; Persistent isolated chemotherapy-induced thrombocytopenia; CIT; Chemotherapy-induced thrombocytopenia; Romiplostim N01; ATRA; All-trans retinoic acid; Tretinoin; Thrombopoietin receptor agonist; Gastrointestinal solid tumor; Digestive system cancer; Pancreatic cancer; Colorectal cancer; Complete remission; Platelet response; Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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.

  From randomization to Week 12

Secondary Outcomes (10)

• Sustained Platelet Response Rate During Weeks 13 to 24

  Weeks 13 through 24

• Complete Response Rate

  Up to Week 24

• Partial Response Rate

  Up to Week 24

• Duration of Response

  From first documented response to Week 24

• Time to Response

  From treatment start to Week 24

Study Arms (2)

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.

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.

Drug: Romiplostim N01

Interventions

Romiplostim N01 DRUG

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.

Also known as: N01, TPO receptor agonist, Thrombopoietin receptor agonist

Romiplostim N01 Alone; Romiplostim N01 Plus ATRA

All-trans retinoic acid DRUG

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.

Also known as: ATRA, Tretinoin

Romiplostim N01 Plus ATRA

Eligibility Criteria

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

You may qualify if:

• Age 18 years or older.
• Prior diagnosis of a gastrointestinal/digestive system solid tumor, including but not limited to gastrointestinal tract, pancreatic, or colorectal cancer.
• 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.
• 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.
• Women of childbearing potential must have a negative pregnancy test before enrollment and agree to use effective contraception during study treatment.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Peking University Cancer Hospital and Institute: collaborator
• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: collaborator
• Peking University International Hospital: collaborator
• Shanxi Province Cancer Hospital: collaborator
• Henan Provincial People's Hospital: collaborator
• China-Japan Union Hospital, Sun Yat-sen University Cancer Center: collaborator
• Hebei Medical University Fourth Hospital: collaborator
• Tianjin Medical University Cancer Institute and Hospital: collaborator

Study Sites (1)

Peking University People's Hospital

Beijing, 100044, China

RECRUITING

Related Publications (5)

• Gao A, Zhang L, Zhong D. Chemotherapy-induced thrombocytopenia: literature review. Discov Oncol. 2023 Jan 25;14(1):10. doi: 10.1007/s12672-023-00616-3.

  PMID: 36695938 BACKGROUND

• Yang J, Zhao L, Wang W, Wu Y. All-trans retinoic acid added to treatment of primary immune thrombocytopenia: a systematic review and meta-analysis. Ann Hematol. 2023 Jul;102(7):1695-1704. doi: 10.1007/s00277-023-05263-w. Epub 2023 May 11.

  PMID: 37166528 BACKGROUND

• Vianelli N, Auteri G, Buccisano F, Carrai V, Baldacci E, Clissa C, Bartoletti D, Giuffrida G, Magro D, Rivolti E, Esposito D, Podda GM, Palandri F. Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives. Ann Hematol. 2022 May;101(5):963-978. doi: 10.1007/s00277-022-04786-y. Epub 2022 Feb 24.

  PMID: 35201417 BACKGROUND

• Soff GA, Al-Samkari H, Leader A, Eisen M, Saad H. Romiplostim in chemotherapy-induced thrombocytopenia: A review of the literature. Cancer Med. 2024 Aug;13(15):e7429. doi: 10.1002/cam4.7429.

  PMID: 39135303 BACKGROUND

• Kuter DJ. Treatment of chemotherapy-induced thrombocytopenia in patients with non-hematologic malignancies. Haematologica. 2022 Jun 1;107(6):1243-1263. doi: 10.3324/haematol.2021.279512.

  PMID: 35642485 BACKGROUND

MeSH Terms

Conditions

Gastrointestinal Neoplasms; Pancreatic Neoplasms; Colorectal Neoplasms; Digestive System Neoplasms; Pathologic Complete Response; Thrombocytopenia

Interventions

Tretinoin

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Endocrine Gland Neoplasms; Pancreatic Diseases; Endocrine System Diseases; Intestinal Neoplasms; Colonic Diseases; Intestinal Diseases; Rectal Diseases; Disease Progression; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Intervention Hierarchy (Ancestors)

Vitamin A; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Diterpenes; Pigments, Biological; Biological Factors

Study Officials

• Xiaohui Zhang, MD

  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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaohui Zhang, MD

CONTACT

+8610-8832-4672; zhangxh@bjmu.edu.cn

Kexin Shen, MD

CONTACT

+8617710635036; keceyshen@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking University Institute of Hematology

Model Details: Eligible adult participants with persistent isolated chemotherapy-induced thrombocytopenia after complete remission of gastrointestinal/digestive system 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.

Study Record Dates

• First Submitted: May 7, 2026
• First Posted: May 14, 2026
• Study Start: December 22, 2025
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: May 14, 2026

Record last verified: 2026-05

Locations

CN (1)