Status:
COMPLETED
A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Aplastic Anemia
Neutropenia
Eligibility:
All Genders
2+ years
Phase:
PHASE1
PHASE2
Brief Summary
Background: * Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard initial treatment for SAA in...
Detailed Description
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow. Allogeneic hematopoietic stem cell transplantation (HSCT) ...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Severe aplastic anemia characterized by:
- Bone marrow cellularity \< 30 percent (excluding lymphocytes)
- AND
- At least two of the following:
- Absolute neutrophil count \< 500/ microL
- Platelet count \< 20,000/ microL
- Absolute reticulocyte count \< 60,000/ microL
- Failure to respond to an initial course of h-ATG/CsA at least 3 months post-treatment or a suboptimal response to initial h-ATG/CsA defined by both platelet and reticulocyte count \< 50,000 /microL at 3 months post-treatment
- OR
- Refractory SAA unresponsive to both horse and rabbit ATG-based regimens
- Age greater than or equal to 2 years old
- Weight greater than or equal to 12 kg
- EXCLUSION CRITERIA:
- Diagnosis of Fanconi anemia
- Cardiac ejection fraction \< 30 percent (evaluated by ECHO)
- Evidence of a clonal hematologic bone marrow disorder on cytogenetics. Patients with the presence of trisomy 8, loss of Y or del(20q) will not be excluded in the absence of dysplastic changes in the marrow. Patients with very severe neutropenia (ANC \< 200 /microL) will not be excluded initially if cytogenetics are not available or pending. If evidence of a clonal disorder is later identified, the patient will go off study)
- Prior immunosuppressive therapy with high dose Cy
- Infection not adequately controlled with appropriate therapy
- Serologic evidence of HIV infection
- 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 30 days is likely
- Subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects
- Current pregnancy or unwillingness to take oral contraceptives or refrain from pregnancy if of childbearing potential
- Not able to understand the investigational nature of the study or to give informed consent
Exclusion
Key Trial Info
Start Date :
August 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2012
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT01187017
Start Date
August 1 2010
End Date
July 1 2012
Last Update
July 2 2021
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892