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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01443351
Recruitment Status : Recruiting
First Posted : September 29, 2011
Last Update Posted : February 5, 2015
Rigshospitalet, Denmark
Odense University Hospital
Information provided by (Responsible Party):
Sif Gudbrandsdottir, Zealand University Hospital

Brief Summary:
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.

Condition or disease
Idiopathic Thrombocytopenic Purpura

Study Type : Observational
Estimated Enrollment : 50 participants
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)
Study Start Date : March 2012
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : March 2020

ITP patients
Patients with refractory ITP eligible for treatment with TPO-ra

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

Secondary Outcome Measures :
  1. Yearly response rates [ Time Frame: 5 years ]
    Number of patients with response to treatment at 1, 2, 3, 4, and 5-year follow-up

  2. Frequency of relapse [ Time Frame: 5 years ]
  3. Rate of splenectomy [ Time Frame: 5 years ]
  4. Development of reticulin fibrosis in bone marrow biopsies [ Time Frame: 5 years ]

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

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): 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
Zealand University 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

Responsible Party: Sif Gudbrandsdottir, MD, Zealand University Hospital Identifier: NCT01443351     History of Changes
Other Study ID Numbers: TPO-SJ-227
First Posted: September 29, 2011    Key Record Dates
Last Update Posted: February 5, 2015
Last Verified: February 2015

Keywords provided by Sif Gudbrandsdottir, Zealand University 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