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

Immune Thrombocytopenic Purpura Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01652599
• Other study ID #: HE12-010
• Status: Completed
• Phase: Phase 2
• First received: July 26, 2012
• Last updated: January 3, 2014
• Start date: June 2012
• Est. completion date: November 2013

Study information

• Verified date: January 2014
• Source: Hospital Universitario Dr. Jose E. Gonzalez
• Contact: n/a
• Is FDA regulated: No
• Health authority: Mexico: Ethics Committee
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: November 2013
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 85 Years

Eligibility

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

Exclusion Criteria:

• Performance status above or equal to 2.

• Previous treatment with eltrombopag

• Immunosuppressive treatment within the last month

• Previous splenectomy

• Presence of malignant haematological disease

• Connective tissue disease

• Autoimmune hemolytic anemia

• Pregnancy and lactation

• Not willing to participate in the study.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Immune Thrombocytopenic Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic

Intervention

Drug:
• Eltrombopag and dexamethasone
Eltrombopag 50 mg PO days 5-32 Dexamethasone 40 mg PO days 1-4

Locations

Country	Name	City	State
Mexico	Hospital Universitario "Dr. Jose E. Gonzalez" UANL	Monterrey	Nuevo Leon

Sponsors (1)

Lead Sponsor	Collaborator
Hospital Universitario Dr. Jose E. Gonzalez

Country where clinical trial is conducted

Mexico, 

References & Publications (2)

Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. doi: 10.1016/S0140-6736(09)60402-5. — View Citation

Jenkins JM, Williams D, Deng Y, Uhl J, Kitchen V, Collins D, Erickson-Miller CL. Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist. Blood. 2007 Jun 1;109(11):4739-41. Epub 2007 Feb 27. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of patients with sustained response after 6 months	Number of patients with complete response at month 6	6 months	No
Secondary	Number of patients with complete response at month 6	Number of patients with platelet count at least 150x109/L, 6 months after therapy	month 6	No
Secondary	Bleeding	Number of patients with bleeding complication therapy	month 6	Yes