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Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Immune Thrombocytopenia (ITP)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2015 by Roskilde County Hospital
Rigshospitalet, Denmark
Odense University Hospital
Information provided by (Responsible Party):
Sif Gudbrandsdottir, Roskilde County Hospital Identifier:
First received: September 13, 2011
Last updated: February 4, 2015
Last verified: February 2015
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatment modalities for patients with refractory Primary Immune Thrombocytopenia (ITP), but only few data are available for long-term effects of these drugs. In this observational study, effects and adverse effects including evaluation of bone marrow biopsies done at fixed intervals will be recorded from ITP patients treated with TPO-ra. For some patients, blood samples will be collected for research use.

Idiopathic Thrombocytopenic Purpura

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Primary Immune Thrombocytopenia (ITP)

Resource links provided by NLM:

Further study details as provided by Roskilde County Hospital:

Primary Outcome Measures:
  • Number of patients with sustained response at 6 months follow-up [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Response is defined as platelet count > 30.000 mio/L and no bleeding

Secondary Outcome Measures:
  • Yearly response rates [ Time Frame: 5 years ] [ Designated as safety issue: No ]
    Number of patients with response to treatment at 1, 2, 3, 4, and 5-year follow-up

  • Frequency of relapse [ Time Frame: 5 years ] [ Designated as safety issue: No ]
  • Rate of splenectomy [ Time Frame: 5 years ] [ Designated as safety issue: No ]
  • Development of reticulin fibrosis in bone marrow biopsies [ Time Frame: 5 years ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA
Whole blood samples will be collected, seru, plasma and mononuclear cells will be stored in a biobank for further research use

Estimated Enrollment: 50
Study Start Date: March 2012
Estimated Study Completion Date: March 2020
Estimated Primary Completion Date: March 2020 (Final data collection date for primary outcome measure)
ITP patients
Patients with refractory ITP eligible for treatment with TPO-ra


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patinets will be selected from Departments of Hematology

Inclusion Criteria:

  • Adult patients with ITP
  • platelet counts < 25 x10*9/L or < 50 x10*9/L with bleeding symptoms
  • meets criteria for treatment with TPO-ra
  • Females must use contraceptives when applicable for at least three months before inclusion

Exclusion Criteria:

  • Pregnancy or nursing
  • Former thromboembolic events excluding one incidence of deep venous thrombosis as complication to surgery or pregnancy or one incidence of cerebral embolism as complication to atrial fibrillation
  • Liver insufficiency (for eltrombopag only)
  • TPO-ra contraindications (e.g. allergy)
  • TPO-ra treatments less than 6 months prior to inclusion
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01443351

Contact: Hans C Hasselbalch, Prof., MD +45 26223678
Contact: Sif Gudbrandsdottir, MD +45 22270982

Copenhagen University Hospital Roskilde Recruiting
Roskilde, Denmark, 4000
Contact: Hans C Hasselbalch, Prof., MD    +45 26223678   
Principal Investigator: Hans C Hasselbalch, Prof., MD         
Sponsors and Collaborators
Roskilde County Hospital
Rigshospitalet, Denmark
Odense University Hospital
Principal Investigator: Hans Hasselbalch, Prof., MD Department of Hematology, Copenhagen University Hospital Roskilde
Principal Investigator: Henrik Frederiksen, Dr., MD Odense University Hospital
Principal Investigator: Sif Gudbrandsdottir, MD Copenhagen University Hospital Roskilde
  More Information

Responsible Party: Sif Gudbrandsdottir, MD, Roskilde County Hospital Identifier: NCT01443351     History of Changes
Other Study ID Numbers: TPO-SJ-227 
Study First Received: September 13, 2011
Last Updated: February 4, 2015
Health Authority: Denmark: The Danish National Committee on Biomedical Research Ethics

Keywords provided by Roskilde County Hospital:
bone marrow fibrosis
long-term safety

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Blood Coagulation Disorders
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases processed this record on October 21, 2016