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.

Conditions

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

Interventions

DRUG

Romiplostim N01

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.

DRUG

All-trans retinoic acid

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.

Sponsors & Collaborators

• Peking University Cancer Hospital and Institute

  collaborator OTHER

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences

  collaborator OTHER

• Peking University International Hospital

  collaborator OTHER

• Shanxi Province Cancer Hospital

  collaborator OTHER

• Henan Provincial People's Hospital

  collaborator OTHER

• China-Japan Union Hospital, Sun Yat-sen University Cancer Center

  collaborator UNKNOWN

• Hebei Medical University Fourth Hospital

  collaborator OTHER

• Tianjin Medical University Cancer Institute and Hospital

  collaborator OTHER

• Peking University People's Hospital

  lead OTHER

Principal Investigators

• 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

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-12-22

Primary Completion

2027-10-31

Completion

2027-12-31

Countries

• China

Study Locations