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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
David Gomez Almaguer, Hospital Universitario Dr. Jose E. Gonzalez
ClinicalTrials.gov Identifier:
NCT01652599
First received: July 26, 2012
Last updated: January 3, 2014
Last verified: January 2014
  Purpose

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


Condition Intervention Phase
Immune Thrombocytopenic Purpura
Drug: Eltrombopag and dexamethasone
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Eltrombopag and High-dose Dexamethasone as First Line Treatment for Immune Thrombocytopenia

Resource links provided by NLM:


Further study details as provided by Hospital Universitario Dr. Jose E. Gonzalez:

Primary Outcome Measures:
  • Number of patients with sustained response after 6 months [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Number of patients with complete response at month 6


Secondary Outcome Measures:
  • Number of patients with complete response at month 6 [ Time Frame: month 6 ] [ Designated as safety issue: No ]
    Number of patients with platelet count at least 150x109/L, 6 months after therapy

  • Bleeding [ Time Frame: month 6 ] [ Designated as safety issue: Yes ]
    Number of patients with bleeding complication therapy


Estimated Enrollment: 30
Study Start Date: June 2012
Study Completion Date: November 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eltrombopag and dexamethasone Drug: Eltrombopag and dexamethasone
Eltrombopag 50 mg PO days 5-32 Dexamethasone 40 mg PO days 1-4
Other Name: Revolade

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.

  Eligibility

Ages Eligible for Study:   18 Years to 85 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01652599

Locations
Mexico
Hospital Universitario "Dr. Jose E. Gonzalez" UANL
Monterrey, Nuevo Leon, Mexico, 64460
Sponsors and Collaborators
Hospital Universitario Dr. Jose E. Gonzalez
Investigators
Principal Investigator: David Gomez-Almaguer, MD Hospital Universitario Dr. Jose E. Gonzalez
  More Information

Publications:
Responsible Party: David Gomez Almaguer, MD, Hospital Universitario Dr. Jose E. Gonzalez
ClinicalTrials.gov Identifier: NCT01652599     History of Changes
Other Study ID Numbers: HE12-010
Study First Received: July 26, 2012
Last Updated: January 3, 2014
Health Authority: Mexico: Ethics Committee

Keywords provided by Hospital Universitario Dr. Jose E. Gonzalez:
Eltrombopag
Dexamethasone
High-dose
ITP

Additional relevant MeSH terms:
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases
Dexamethasone acetate
Dexamethasone
Dexamethasone 21-phosphate
BB 1101
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Central Nervous System Agents
Gastrointestinal Agents
Glucocorticoids
Hormones

ClinicalTrials.gov processed this record on July 28, 2014