Back to resultsUsing ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell (AGRAH003)
Primary Purpose
Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Status
Completed
Phase
Phase 1
Locations
France
Study Type
Interventional
Intervention
Romiplostim
Sponsored by
About this trial
This is an interventional treatment trial for Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Eligibility 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.
- 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
Sites / Locations
- Saint Louis hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Romiplostim
Arm Description
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
Outcomes
Primary Outcome Measures
Adverse events
Incidence and severity of all adverse events
Secondary Outcome Measures
Dose of Romiplostim
Dose of Romiplostim required to reach a platelet count above 50 x 109/L in absence of platelet transfusion
Durable platelet response after transplant:
platelet count above 50 x 109/L on 8 consecutive weeks independent of platelet transfusions
Relapse rate
Graft versus host disease (GVHD)
Non relapse mortality rate
number of platelet transfusions
Overall number of bleeding events
platelet hematological improvements
Incidence and duration of platelet hematological improvements above 20 x 109/L and above 50 x 109/L , respectively
Full Information
NCT ID
NCT01980030
First Posted
November 4, 2013
Last Updated
February 13, 2019
Sponsor
Assistance Publique - Hôpitaux de Paris
1. Study Identification
Unique Protocol Identification Number
NCT01980030
Brief Title
Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
Acronym
AGRAH003
Official Title
Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
Study Type
Interventional
2. Study Status
Record Verification Date
February 2019
Overall Recruitment Status
Completed
Study Start Date
November 2013 (undefined)
Primary Completion Date
December 2015 (Actual)
Study Completion Date
November 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris
4. Oversight
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
24 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Romiplostim
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Romiplostim
Primary Outcome Measure Information:
Title
Adverse events
Description
Incidence and severity of all adverse events
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Dose of Romiplostim
Description
Dose of Romiplostim required to reach a platelet count above 50 x 109/L in absence of platelet transfusion
Time Frame
12 months
Title
Durable platelet response after transplant:
Description
platelet count above 50 x 109/L on 8 consecutive weeks independent of platelet transfusions
Time Frame
12 months
Title
Relapse rate
Time Frame
12 months
Title
Graft versus host disease (GVHD)
Time Frame
12 months
Title
Non relapse mortality rate
Time Frame
12 months
Title
number of platelet transfusions
Time Frame
12 months
Title
Overall number of bleeding events
Time Frame
12 months
Title
platelet hematological improvements
Description
Incidence and duration of platelet hematological improvements above 20 x 109/L and above 50 x 109/L , respectively
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
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
Facility Information:
Facility Name
Saint Louis hospital
City
Paris
State/Province
Ile De France
ZIP/Postal Code
75010
Country
France
12. IPD Sharing Statement
Learn more about this trial
Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
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