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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.
Verified March 2012 by Roskilde County Hospital
Sponsor:
Collaborators:
Rigshospitalet, Denmark
Odense University Hospital
Information provided by (Responsible Party):
Sif Gudbrandsdottir, Roskilde County Hospital
ClinicalTrials.gov Identifier:
NCT01443351
First received: September 13, 2011
Last updated: March 15, 2012
Last verified: March 2012
History of Changes
  Purpose

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
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:

U.S. FDA Resources

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)
Groups/Cohorts
ITP patients
Patients with refractory ITP eligible for treatment with TPO-ra

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patinets will be selected from Departments of Hematology

Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01443351

Contacts
Contact: Hans C Hasselbalch, Prof., MD +45 26223678 hans.hasselbalch@dadlnet.dk
Contact: Sif Gudbrandsdottir, MD +45 22270982 sif.gudbrandsdottir@gmail.com

Locations
Denmark
Copenhagen University Hospital Roskilde Recruiting
Roskilde, Denmark, 4000
Contact: Hans C Hasselbalch, Prof., MD     +45 26223678     hans.hasselbalch@dadlnet.dk    
Principal Investigator: Hans C Hasselbalch, Prof., MD            
Sponsors and Collaborators
Roskilde County Hospital
Rigshospitalet, Denmark
Odense University Hospital
Investigators
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

No publications provided

Responsible Party: Sif Gudbrandsdottir, MD, Roskilde County Hospital
ClinicalTrials.gov Identifier: NCT01443351     History of Changes
Other Study ID Numbers: TPO-SJ-227
Study First Received: September 13, 2011
Last Updated: March 15, 2012
Health Authority: Denmark: The Danish National Committee on Biomedical Research Ethics

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

Additional relevant MeSH terms:
Purpura
Purpura, Thrombocytopenic
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
 Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases

ClinicalTrials.gov processed this record on May 21, 2013