AGRAH003: Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 1/Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: 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 |
Drug: Romiplostim
|
Outcome Measures
Primary Outcome Measures
- Adverse events [12 months]
Incidence and severity of all adverse events
Secondary Outcome Measures
- Dose of Romiplostim [12 months]
Dose of Romiplostim required to reach a platelet count above 50 x 109/L in absence of platelet transfusion
- 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]
- 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
Eligibility Criteria
Criteria
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.
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Prolonged (> 2 months) transfusion-dependent thrombocytopenia
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Screenings mean platelet count≤ 20 x giga/L or screenings mean platelet count ≤ 50 x giga/L with a history of bleeding.
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(ECOG) performance status of 0-2
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Adequate liver function
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Serum creatinine ≤ 176.8 μmol/L
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Bone marrow aspirate with cytogenetics within 6 days of the first dose of romiplostim
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Written informed consent
Exclusion Criteria:
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Relapse/progression of hematological malignancy (marrow examination required)
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Non-controlled acute and/or chronic graft versus host disease (GvHD)
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Active or uncontrolled infections
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Cardiac pathology - Thrombosis
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Pregnancy or breast feeding
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Received interleukin-11 (IL-11) within 4 weeks of screening or previously received any thrombopoietic growth factor
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Patients on anticoagulant therapy
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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
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Subject not using adequate contraceptive precautions, in the judgment of the investigator
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Sensitivity to any Escherichia coli-derived product
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Inability to comply with study procedures.
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Subject currently is enrolled in or has not yet completed 30 days since ending other investigational device or drug study
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No medical insurance in the French Health system
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Saint Louis hospital | Paris | Ile De France | France | 75010 |
Sponsors and Collaborators
- Assistance Publique - Hôpitaux de Paris
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- P110204