Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
Trial record 2 of 3 for:    immune thrombocytopenia OR immune thrombocytopenic purpura OR idiopathic thrombocytopenic purpura | Open Studies | NIH, U.S. Fed

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2015 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier:
NCT01623167
First received: June 14, 2012
Last updated: February 7, 2015
Last verified: January 2015
  Purpose

Background:

  • Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment.
  • Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts.

Objectives:

- To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.

Eligibility:

- Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated.

Design:

  • Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected.
  • Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease.
  • After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months.
  • Participants may receive other medications to prevent infections during treatment.
  • Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality of life.

Condition Intervention Phase
Aplastic Anemia
Neutropenia
Pancytopenia
Anemia
Thrombocytopenia
Drug: Eltrombopag
Drug: Horse Anti-Thymocyte Globulin (ATG)
Drug: Cyclosporine A (CSA)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • The primary endpoint will be the rate of complete hematologic response at six months.

Secondary Outcome Measures:
  • Secondary endpoints are relapse, robust hematologic blood count recovery at 3, 6, and 12 months, survival, clonal evolution to myelodysplasia and leukemia, and marrow stem cell content.

Estimated Enrollment: 95
Study Start Date: June 2012
Estimated Study Completion Date: January 2021
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Eltrombopag
    N/A
    Drug: Horse Anti-Thymocyte Globulin (ATG)
    N/A
    Drug: Cyclosporine A (CSA)
    N/A
  Show Detailed Description

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

-INCLUSION CRITERIA:

  1. Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes)

    AND

    At least two of the following:

    • Absolute neutrophil count less than 500/microL
    • Platelet count less than 20,000/microL

    Absolute reticulocyte count less than 60,000/microL

  2. Age greater than or equal to 2 years old
  3. Weight greater than 12 kg

EXCLUSION CRITERIA:

  1. Known diagnosis of Fanconi anemia
  2. Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry. Patients with super severe neutropenia (ANC less than 200 /microL) will not be excluded initially if cytogenetics are not available or pending. If evidence of a clonal disorder consistent with myelodysplasia is later identified, the patient will go off study.
  3. Prior immunosuppressive therapy with any ATG, alemtuzumab, or high dose cyclophosphamide
  4. SGOT or SGPT greater than 3 times the upper limit of normal
  5. Subjects with liver cirrhosis
  6. Hypersensitivity to eltrombopag or its components
  7. Infection not adequately responding to appropriate therapy
  8. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy, or that death within 7-10 days is likely
  9. Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
  10. Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of this study
  11. Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent per section 11.6.
  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: NCT01623167

Contacts
Contact: Olga J Rios, R.N. (301) 496-4462 olga.rios@nih.gov
Contact: Danielle M Townsley, M.D. (301) 402-3477 townsleydm@nhlbi.nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010    prpl@mail.cc.nih.gov   
Sponsors and Collaborators
Investigators
Principal Investigator: Danielle M Townsley, M.D. National Heart, Lung, and Blood Institute (NHLBI)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01623167     History of Changes
Other Study ID Numbers: 120150, 12-H-0150
Study First Received: June 14, 2012
Last Updated: February 7, 2015
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
T-cells
Immunosuppression
Hematopoesis
Autoimmunity
Thrombocytopenia

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Thrombocytopenia
Blood Platelet Disorders
Bone Marrow Diseases
Hematologic Diseases

ClinicalTrials.gov processed this record on March 01, 2015