This trial is active, not recruiting.

Condition persistent thrombocytopenia following allogeneic hematopoietic stem cell transplantation (hsct)
Treatment romiplostim
Phase phase 1/phase 2
Sponsor Assistance Publique - Hôpitaux de Paris
Start date November 2013
End date December 2015
Trial size 24 participants
Trial identifier NCT01980030, P110204


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
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 Outcomes

Adverse events
time frame: 12 months

Secondary Outcomes

Dose of Romiplostim
time frame: 12 months
Durable platelet response after transplant:
time frame: 12 months
Relapse rate
time frame: 12 months
Graft versus host disease (GVHD)
time frame: 12 months
Non relapse mortality rate
time frame: 12 months
number of platelet transfusions
time frame: 12 months
Overall number of bleeding events
time frame: 12 months
platelet hematological improvements
time frame: 12 months

Eligibility Criteria

Male or female participants at least 18 years old.

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

Additional Information

Official title Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Assistance Publique - Hôpitaux de Paris.