Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell (AGRAH003)

Assistance Publique - Hôpitaux de Paris

Status and phase: Completed; Phase 2; Phase 1

Conditions: Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Treatments: Drug: Romiplostim

Study type: Interventional

Funder types: Other

Identifiers

NCT01980030

P110204

Details and patient eligibility

About

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.

Enrollment: 24 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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24 participants in 1 patient group

Romiplostim

Experimental group

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

Treatment:

Drug: Romiplostim