Status:
COMPLETED
A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Anemia, Aplastic
Anemia, Hypoplastic
Eligibility:
All Genders
12+ years
Phase:
PHASE2
Brief Summary
Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patie...
Detailed Description
Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patie...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Diagnosis of aplastic anemia, with refractory thrombocytopenia following at least one treatment course of horse or rabbit ATG/cyclosporine.
- Platelet count less than or equal to 30,000/microL
- Age greater than or equal to 12 years old
- EXCLUSION CRITERIA:
- Diagnosis of Fanconi anemia
- Infection not adequately responding to appropriate therapy
- Patients with a PNH clone size in neutrophils of greater than or equal to 50%
- HIV positivity
- Creatinine \> 2.5
- Bilirubin \> 2.0
- SGOT or SGPT \> 5 times the upper limit of normal
- Hypersensitivity to eltrombopag or its components
- Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
- History of malignancy other than localized tumors diagnosed more than one year previously and treated surgically with curative intent (for instance squamous cell or other skin cancers, stage 1 breast cancer, cervical carcinoma in situ, etc)
- Unable to understand the investigational nature of the study or give informed consent
- History of congestive heart failure arrhythmia requiring chronic treatment, arterial or venous thrombosis (not excluding line thrombosis) within the last 1 year, or myocardial infarction within 3 months before enrollment
- ECOG Performance Status of 3 or greater
- Treatment with horse or rabbit ATG or Campath within 6 months of study entry. Concurrent stable treatment with cyclosporine or G-CSF is permitted.
Exclusion
Key Trial Info
Start Date :
May 29 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 24 2018
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT00922883
Start Date
May 29 2009
End Date
April 24 2018
Last Update
October 20 2020
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892