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Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia | Clinical Trials | Clareo Health

COMPLETEDPHASE2

Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

NCT01703169•University of Utah

View on ClinicalTrials.gov

Summary

The investigators hypothesis is that eltrombopag given to patients with moderate to very severe aplastic anemia will result in an increase in platelet counts. The investigators hypothesize that in patients with moderate to very severe aplastic anemia, treatment with eltrombopag will lead to fewer platelet transfusions, red blood cell transfusions, and fewer bleeding events. The investigators hypothesize that in patients with moderate to very severe aplastic anemia, eltrombopag will have an acceptable toxicity rate \<3%, at doses that result in increased platelet counts. Finally the investigators hypothesize that plasma eltrombopag levels in peripheral blood will correlate with improved platelet counts.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Able to provide written informed consent and any other authorizations required by local law (e.g., Protected Health Information \[PHI\])
•Have severe or very severe aplastic anemia, or moderate aplastic anemia with platelet counts that have dropped below 20,000/μl
•Have moderate, severe, or very severe aplastic anemia with moderate bleeding during or after a surgical procedure, (including bone marrow biopsy, lumbar puncture, thoracentesis, paracentesis, port placement, dermal biopsy) or minimal mucocutaneous bleeding otherwise noted
•Subjects with current or previous exposure to approved medications for the treatment of aplastic anemia will not be excluded; these include but may not be limited to, anti-thymocyte globulin (ATG), cyclosporine, corticosteroids, and G-CSF.

Exclusion Criteria

•Have diagnosis of Fanconi anemia
•Have infection not adequately responding to appropriate therapy
•Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50%
•Have known HIV positivity
•Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal
•Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with ATG within three weeks of screening.
•Have AST and/or ALT ≥ 3 times the upper limit of normal
•Have hypersensitivity to eltrombopag or its components
•Have chemotherapy given less than or equal to 14 days prior to initiating the study medication. This does not include immunosuppressive agents and growth factor as described above
•Are female and are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
+6 more criteria

Locations (1)

University of Utah

Salt Lake City, Utah, United States

Key Dates

Start Date

November 1, 2012

Primary Completion Date

June 1, 2016

Completion Date

June 1, 2016

Last Updated

October 19, 2017

Enrollment

ACTUAL participants

Conditions

Severe Aplastic AnemiaVery Severe Aplastic AnemiaModerate Aplastic Anemia

Interventions

Eltrombopag

DRUG

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05012111

Severe Aplastic AnemiaTelomere Biology Disorders+1 more

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RECRUITINGPHASE2

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)+1 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 19, 2017Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

Inclusion Criteria

Exclusion Criteria

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

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