Clinical trial NCT01980030: Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
Official title: Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2013-11-08
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Adverse events
timeFrame: 12 months
Secondary outcomes:
measure: Dose of Romiplostim
timeFrame: 12 months
measure: Durable platelet response after transplant:
timeFrame: 12 months
measure: Relapse rate
timeFrame: 12 months
measure: Graft versus host disease (GVHD)
timeFrame: 12 months
measure: Non relapse mortality rate
timeFrame: 12 months
measure: number of platelet transfusions
timeFrame: 12 months
measure: Overall number of bleeding events
timeFrame: 12 months
measure: platelet hematological improvements
timeFrame: 12 months
Other outcomes:
Locations:
city: Paris
country: France
facility: Saint Louis hospital
state: Ile De France
Study officials:
References: