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

NCT01980030 | Completed Phase 1 DMC

• 1 other identifier: P110204
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Persistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Outcome Measures

Primary Outcomes (1)

• Adverse events

  Incidence and severity of all adverse events

  12 months

Secondary Outcomes (8)

• Dose of Romiplostim

  12 months

• Durable platelet response after transplant:

  12 months

• Relapse rate

  12 months

• Graft versus host disease (GVHD)

  12 months

• Non relapse mortality rate

  12 months

Study Arms (1)

Romiplostim

EXPERIMENTAL

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

Interventions

Romiplostim DRUG

Romiplostim

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Saint Louis hospital

Paris, Île-de-France Region, 75010, France

Location

MeSH Terms

Interventions

romiplostim

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 4, 2013
• First Posted: November 8, 2013
• Study Start: November 1, 2013
• Primary Completion: December 1, 2015
• Study Completion: November 1, 2018
• Last Updated: February 15, 2019

Record last verified: 2019-02

Locations

FR (1)