Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
- Conditions
- Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- Interventions
- Registration Number
- NCT01980030
- Lead Sponsor
- Assistance Publique - Hôpitaux de Paris
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 24
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Romiplostim Romiplostim 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
- Primary Outcome Measures
Name Time Method Adverse events 12 months Incidence and severity of all adverse events
- Secondary Outcome Measures
Name Time Method Dose of Romiplostim 12 months Dose of Romiplostim required to reach a platelet count above 50 x 109/L in absence of platelet transfusion
number of platelet transfusions 12 months Durable platelet response after transplant: 12 months platelet count above 50 x 109/L on 8 consecutive weeks independent of platelet transfusions
Relapse rate 12 months Graft versus host disease (GVHD) 12 months Non relapse mortality rate 12 months Overall number of bleeding events 12 months platelet hematological improvements 12 months Incidence and duration of platelet hematological improvements above 20 x 109/L and above 50 x 109/L , respectively
Trial Locations
- Locations (1)
Saint Louis hospital
🇫🇷Paris, Ile De France, France