Eltrombopag and High-dose Dexamethasone as First Line Treatment for IT

Sponsor

Hospital Universitario Dr. Jose E. Gonzalez (Other)

Overall Status

Completed

CT.gov ID

NCT01652599

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

1.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to determine the response rate and response duration with the combination of eltrombopag and high-dose dexamethasone

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenic Purpura	Drug: Eltrombopag and dexamethasone	Phase 2

Detailed Description

Immune Thrombocytopenia is an autoimmune disorder characterized by formation of autoantibodies against platelet antigens leading platelet destruction.

Corticosteroids increase the platelet count in about 80 percent of patients.However, many patients have a relapse when the dose of corticosteroid is reduced. Debilitating side effects are common in patients who require long-term corticosteroid therapy to maintain the platelet count. Eltrombopag, it is a small molecule agonist of the c-mpl (TpoR) receptor, which is the physiological target of the hormone thrombopoietin, has been shown to be effectively raise the platelet count in adult patients (aged 18 years and over) who have had their spleen removed or where splenectomy is not an option and have received prior treatment with corticosteroids or immunoglobulins, and these medicines did not work (refractary ITP). There are a few case reports where eltrombopag was an option as first line treatment for IT.

The purpose of this study is to determine the response rate and response duration with the combination of eltrombopag (50mg PO once a day for 4 weeks) and high-dose dexamethasone (40mg PO days 1,2,3,4) in untreated adult patients immune thrombocytopenic or in patients with less than 7 days of treatment with corticosteroids.

A complete platelet response is defined as an increase in platelet counts to >150×109/L on two consecutive occasions. A partial response is defined as an increase in the platelet count to between 50 and 150×109/L on two consecutive occasions, 1 week apart. Duration of response is considered from the day of the initial administration to the first time of relapse (platelet count <30×109/L)or to time of analysis.

At the end of the first 5 weeks, the patients will followed by 6 months every month.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Eltrombopag and High-dose Dexamethasone as First Line Treatment for Immune Thrombocytopenia

Study Start Date :

Jun 1, 2012

Actual Primary Completion Date :

Jun 1, 2013

Actual Study Completion Date :

Nov 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Eltrombopag and dexamethasone	Drug: Eltrombopag and dexamethasone Eltrombopag 50 mg PO days 5-32 Dexamethasone 40 mg PO days 1-4 Other Names: Revolade

Arm

Intervention/Treatment

Experimental: Eltrombopag and dexamethasone

Drug: Eltrombopag and dexamethasone

Eltrombopag 50 mg PO days 5-32 Dexamethasone 40 mg PO days 1-4

Other Names:

Revolade

Outcome Measures

Primary Outcome Measures

Number of patients with sustained response after 6 months [6 months]
Number of patients with complete response at month 6

Secondary Outcome Measures

Number of patients with complete response at month 6 [month 6]
Number of patients with platelet count at least 150x109/L, 6 months after therapy
Bleeding [month 6]
Number of patients with bleeding complication therapy

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Clinically confirmed immune thrombocytopenic (IT) Platelet count less than 30,000/mm3 on two occasions. Platelets >30000/mm3 with bleeding.
Less than seven days taking corticosteroids
Normal to increased numbers of megakaryocytes on bone marrow examination in patients ≥ 60 years
Subject is ≥ 18 years
Subject has signed and dated written informed consent.
No sepsis or fever
No active infection requiring therapy
No active chronic viral infection
HIV negative
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception