Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell

Study Overview
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Brief Summary
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This is a Phase I/II multicenter study which aims to assess the toxicity profile of Romiplostim in patients with transfusion-dependent thrombocytopenia after allogeneic HSCT. A total of 24 patients with transfusion-dependent thrombocytopenia after allogeneic HSCT will be included. The main endpoint is the incidence and severity of adverse events.

Official Title
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Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell

Conditions
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Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Intervention / Treatment
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* Drug: Romiplostim


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects must be ≥ 18 years, willing and able to sign informed consent Patients could have been transplanted for hematological disorder (malignant or non-malignant) excepted myelodysplastic syndromes patients and had received either a myeloablative or a reduced intensity conditioning. All sources of allogeneic stem cells are allowed. Prolonged (> 2 months) transfusion-dependent thrombocytopenia Screenings mean platelet count≤ 20 x giga/L or screenings mean platelet count ≤ 50 x giga/L with a history of bleeding. (ECOG) performance status of 0-2 Adequate liver function Serum creatinine ≤ 176.8 μmol/L Bone marrow aspirate with cytogenetics within 6 days of the first dose of romiplostim Written informed consent Exclusion Criteria: Relapse/progression of hematological malignancy (marrow examination required) Non-controlled acute and/or chronic graft versus host disease (GvHD) Active or uncontrolled infections Cardiac pathology - Thrombosis Pregnancy or breast feeding Received interleukin-11 (IL-11) within 4 weeks of screening or previously received any thrombopoietic growth factor Patients on anticoagulant therapy Receipt or planned receipt of Pegylated Granulocyte Colony Stimulating Factor (PEG-G-CSF), or Granulocyte macrophage-colony stimulating factor (GM-CSF) within 4 weeks of the first dose of investigational product Subject not using adequate contraceptive precautions, in the judgment of the investigator Sensitivity to any Escherichia coli-derived product Inability to comply with study procedures. Subject currently is enrolled in or has not yet completed 30 days since ending other investigational device or drug study No medical insurance in the French Health system

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Romiplostim<br>Weekly Romiplostim for 12 weeks with intra-patient weekly dose escalation from 1µg/Kg to a maximum dose of 10 µg/Kg with a dose reduction schema in case of platelet overshoot | Drug: Romiplostim<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events | Incidence and severity of all adverse events | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dose of Romiplostim | Dose of Romiplostim required to reach a platelet count above 50 x 109/L in absence of platelet transfusion | 12 months | 
| Durable platelet response after transplant: | platelet count above 50 x 109/L on 8 consecutive weeks independent of platelet transfusions | 12 months | 
| Relapse rate |  | 12 months | 
| Graft versus host disease (GVHD) |  | 12 months | 
| Non relapse mortality rate |  | 12 months | 
| number of platelet transfusions |  | 12 months | 
| Overall number of bleeding events |  | 12 months | 
| platelet hematological improvements | Incidence and duration of platelet hematological improvements above 20 x 109/L and above 50 x 109/L , respectively | 12 months |