Romiplostim in Chemotherapy-Induced Thrombocytopenia

Safety and Efficacy of Romiplostim in Chemotherapy-Induced Thrombocytopenia in Children and Adolescents With Solid Malignancy

Prospective interventional randomized controlled trial to assess safety and efficacy of romiplostim in chemotherapy-induced thrombocytopenia in children and adolescents with solid malignancy

Study Overview

• Status: Recruiting
• Conditions: Chemotherapy-induced Thrombocytopenia; Solid Malignancy
• Intervention / Treatment: Drug: Romiplostim

Detailed Description

Chemotherapy-induced thrombocytopenia (CIT) frequently complicates cancer treatment causing delays in treatment and dose reduction for subsequent administration of cancer-directed treatment.

Romiplostim is a potential agent that can improve platelet counts, allowing the resumption of chemotherapy, decreasing the need for platelet transfusions, and increasing the nadir platelet counts thus improving dose intensity.

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 3

Contacts and Locations

Study Locations

• Egypt
  • Cairo, Egypt, 11566
    • Recruiting
    • Ain Shams University Children's Hospital
    • Contact: Sara S Makkeyah, PhD; Phone Number: +201140105222; Email: smakkeyah@med.asu.edu.eg
    • Contact: Reem M El-Mazloom, MSc; Phone Number: +201002488174; Email: reem.mazloom@med.asu.edu.eg

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Age: 1- 18 years. Patients diagnosed with solid malignancy based on clinical, histopathological features, and /or immunohistochemical staining.

Patients with chemotherapy-induced thrombocytopenia defined as either

• severe thrombocytopenia either clinically with high bleeding score or laboratory by platelet count reaching critical level < 20x10e9/L requiring platelet transfusion at time of chemotherapy cycle nadir.
• delayed recovery of platelet count: a platelet count of less than 100 x10e9/L for 3 weeks from the first day of chemotherapy cycle administration or the previous dose was reduced by >20% due to low platelet count <100×10e9/L.

Exclusion Criteria:

• Patients with second primary neoplasm.
• Patients with relapsed/refractory solid malignancy.
• Presence of primary or metastatic liver cancer.
• History of a prior symptomatic venous thromboembolic event (VTE) or arterial ischemic events.
• Patients with thrombocytopenia due to other etiologies e.g., underlying inherited thrombocytopenia.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Interventional group; Patients with CIT fulfilling the inclusion criteria will receive romiplostim subcutaneously weekly, either at the time of nadir of a delayed chemotherapy cycle or on the first day of the subsequent chemotherapy cycle and continue until the completion of chemotherapy for maximum 12 weeks. Starting dose will be 3 μg/kg with the escalation of the dose weekly by 2 μg/kg (maximum dose 10 μg/kg) when the platelet increment was less than 25 x10e9/L to maintain platelet counts above 75x10e9/L.	Drug: Romiplostim; Weekly subcutaneous injections; Other Names: TPO-RA
No Intervention: Control group; Patients with CIT fulfilling the inclusion criteria randomized to the control group will not receive romiplostim injections. Patients are allowed to received supportive care according to the standard of care protocols.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hematologic response	Platelet count increase to > 75 x 10e9/L, as well as toleration of chemotherapy resumption for at least 8 weeks or two cycles without subsequent chemotherapy dose reduction or dose delay because of recurrent CIT	12 weeks

Collaborators and Investigators

• Sponsor: Ain Shams University

Study record dates

Study Major Dates

• Study Start (Actual): December 17, 2023
• Primary Completion (Estimated): May 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: December 30, 2023
• First Submitted That Met QC Criteria: December 30, 2023
• First Posted (Estimated): January 11, 2024

Study Record Updates

• Last Update Posted (Estimated): January 11, 2024
• Last Update Submitted That Met QC Criteria: December 30, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Platelet Disorders; Neoplasms; Thrombocytopenia
• Other Study ID Numbers: FAMSU MD343/2023

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No