Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Conditions
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.
Interventions
DRUGRomiplostim
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events | 12 months | Incidence and severity of all adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| 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 | — |
| 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 |
| Non relapse mortality rate | 12 months | — |
Countries
France
Outcome results
None listed