Status:
UNKNOWN
Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia
Lead Sponsor:
Office of Rare Diseases (ORD)
Collaborating Sponsors:
Rare Diseases Clinical Research Network
Conditions:
Anemia, Aplastic
Eligibility:
All Genders
21+ years
Phase:
PHASE1
PHASE2
Brief Summary
Aplastic anemia is a rare autoimmune disorder in which the bone marrow production of blood cells is greatly decreased or absent. Symptoms include fatigue, weakness, tiny reddish-purple marks on the sk...
Detailed Description
The most successful treatment for aplastic anemia is bone marrow transplantation. However, few patients are eligible for this procedure. For others, treatment usually consists of immunosuppressive age...
Eligibility Criteria
Inclusion
- Diagnosis of moderate or severe aplastic anemia with bone marrow cellularity of less than 25%
- Falls within one of the following descriptions at the time of the original diagnosis:
- For severe aplastic anemia, fulfills any two of the following three criteria: absolute neutrophil count less than 500/uL; absolute reticulocyte count less than 60,000/uL; and platelet count less than 20,000/uL
- For moderate aplastic anemia, fulfills any two of the following three criteria: absolute neutrophil count less than 1200/ul; hemoglobin less than 8 g/dL with corrected reticulocyte count less than 1%; and platelet count less than 60,000/uL (Note: Participants who have progressed from moderate to severe aplastic anemia prior to study entry will be classified as having severe aplastic anemia)
- Diagnosis of refractory aplastic anemia, as defined by a failure to achieve at least a partial response to ATG within 6 months of treatment. Individuals who had a prior response to ATG but who have relapsed and not responded to salvage ATG are eligible. Individuals with relapsed disease who are not candidates for salvage ATG because they experienced a serious or life-threatening complication prior to ATG are also eligible.
- A Karnofsky performance status of at least 60%
- Adequate organ function, as defined by creatine levels less than 1.5 times the upper limit normal (ULN), and liver function tests (AST, bilirubin) less than 2 times the ULN
- Women of childbearing age must be willing to use effective contraception throughout the study
Exclusion
- Received ATG treatment less than 6 months prior to study entry
- Candidate for related allogeneic stem cell transplantation
- Active uncontrolled infection
- History of myelodysplastic syndrome or bone marrow cytogenetic abnormalities
- History of Fanconi's anemia or other congenital form of aplastic anemia
- Treatment with an investigational agent within 1 month of study entry
- HIV infection
- Pregnant or breastfeeding
Key Trial Info
Start Date :
May 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2009
Estimated Enrollment :
52 Patients enrolled
Trial Details
Trial ID
NCT00319878
Start Date
May 1 2006
End Date
December 1 2009
Last Update
October 7 2008
Active Locations (4)
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1
UCLA Center for Health Sciences
Los Angeles, California, United States, 90095
2
Lee Moffitt Cancer Center
Tampa, Florida, United States, 33606
3
Taussig Cancer Center, Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
4
Penn State University Cancer Center
Hershey, Pennsylvania, United States, 17033