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Protocol Details

Extended Dosing with Eltrombopag in Refractory Severe Aplastic Anemia

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

13-H-0133

Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 2 Years
Max Age: 99 Years

Referral Letter Required

No

Population Exclusion(s)

None

Keywords

Stem Cells;
Hematopoiesis;
Autoimmunity;
Thrombocytopenia;
Neutropenia

Recruitment Keyword(s)

None

Condition(s)

Refractory Severe Aplastic Anemia

Investigational Drug(s)

Eltrombopag

Investigational Device(s)

None

Intervention(s)

Drug: Eltrombopag

Supporting Site

National Heart, Lung and Blood InstituteNational Heart, Lung, and Blood Institute

Background:

- Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia.

Objectives:

- To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts.

Eligibility:

- Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia.

Design:

- Participants will take eltrombopag by mouth once a day for 6 months.

- Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow.

- Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.

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Eligibility

INCLUSION CRITERIA:

-Previous diagnosis of refractory severe aplastic anemia and following at least one treatment course of immunosuppression with a regimen containing antithymocyte globulin, alemtuzumab or cyclophosphamide.

-One or more of the following three clinically-significant cytopenias: platelet count less than or equal to 30,000/micro L or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); neutrophil count less than 500/micro L; hemoglobin less than 9.0 g/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry)

-Age greater than or equal to 2 years old

-Weight > 12 kg

EXCLUSION CRITERIA:

-Known diagnosis of Fanconi anemia

-Infection not adequately responding to appropriate therapy

-Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry.

-Creatinine > 2.5 mg/dL

-Direct Bilirubin > 2.0 mg/dL

-SGOT or SGPT >5 times the upper limit of normal

-Hypersensitivity to eltrombopag or its components<TAB>

-Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential

-Unable to understand the investigational nature of the study or give informed consent

-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

-Treatment with ATG, cyclophophamide or alemtuzamab within 6 months of study entry.


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Citations:

Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19. Epub 2006 Jun 15.

Risitano AM, Maciejewski JP, Green S, Plasilova M, Zeng W, Young NS. In-vivo dominant immune responses in aplastic anaemia: molecular tracking of putatively pathogenetic T-cell clones by TCR beta-CDR3 sequencing. Lancet. 2004 Jul 24-30;364(9431):355-64.

Chen J. Animal models for acquired bone marrow failure syndromes. Clin Med Res. 2005 May;3(2):102-8.

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Contacts:

Principal Investigator

Referral Contact

For more information:

David J. Young, M.D.
National Heart, Lung and Blood Institute (NHLBI)
NIHBC 10 - CRC BG RM 5-3150
10 CENTER DR
BETHESDA MD 20892
(301) 827-7823
david.young2@nih.gov

Ivana Darden, R.N.
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 4-5362
10 Center Drive
Bethesda, Maryland 20892
(301) 827-2988
ivana.darden@nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1
ccopr@nih.gov

Clinical Trials Number:

NCT01891994

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