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

Withdrawn

• Conditions: Idiopathic Thrombocytopenic Purpura

• Registration Number: NCT01443351
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-09-13
• Study First Submitted QC: 2011-09-28
• Study First Posted: 2011-09-29
• Study Start: 2015-03
• Primary Completion: 2019-08
• Study Completion: 2019-08
• Status Verified: 2019-09
• Last Update Submitted: 2019-09-09
• Last Update Posted: 2019-09-11

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of patients with sustained response at 6 months follow-up	6 months	Response is defined as platelet count \> 30.000 mio/L and no bleeding

Secondary Outcome Measures

Name	Time	Method
Yearly response rates	5 years	Number of patients with response to treatment at 1, 2, 3, 4, and 5-year follow-up
Frequency of relapse	5 years
Rate of splenectomy	5 years
Development of reticulin fibrosis in bone marrow biopsies	5 years

Trial Locations

Locations (1)

Copenhagen University Hospital Roskilde; 🇩🇰 Roskilde, Denmark