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

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.

Timeline

Start

2025-12-22

Primary completion

2027-10-31

Completion

2027-12-31

Publications

• Background 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.
• 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.
• 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.
• 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.
• 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.

Drugs

Indications