This study is NOT currently recruiting participants.
Number
05-H-0242
Sponsoring Institute
National Heart, Lung and Blood Institute (NHLBI)
Recruitment Detail
Type: Completed Study; data analyses ongoing Gender: Male & Female Min Age: 2 Max Age: 110
Referral Letter Required
Yes
Population Exclusion(s)
None
Keywords
Immunosuppression; T-cells; Hematopoiesis; Autoimmunity; Thrombocytopenia
Recruitment Keyword(s)
Condition(s)
Severe Aplastic Anemia, Refractory; Severe Aplastic Anemia, Relapse
Investigational Drug(s)
Alemtuzumab (Campath )
Investigational Device(s)
Intervention(s)
Biological/Vaccine: Alemtuzumab (Campath ) Drug: Cyclosporine
Supporting Site
National Heart, Lung, and Blood Institute
Patients 2 years of age and older with severe aplastic anemia whose disease does not respond to immunosuppressive therapy or has recurred following immunosuppressive therapy may be eligible for this study. Participants undergo the following tests and procedures:
-Pretreatment evaluation: Patients have a medical history, physical examination, blood tests, electrocardiogram (EKG), echocardiogram, 24-hour Holter monitor (continuous 24-hour monitoring of electrical activity of the heart), bone marrow biopsy (withdrawal through a needle of a small sample of bone marrow for analysis).
-Placement of a central line, if needed: An intravenous line (tube) is placed into a major vein in the patient's chest. It can stay in the body for the entire treatment period and be used to give chemotherapy or other medications, including antibiotics and blood transfusions, if needed, and to withdraw blood samples.
-Alemtuzumab therapy: Patients are admitted to the NIH Clinical Center for the first few injections for close monitoring of side effects. After receiving an initial small test dose, patients begin the first of ten daily injections under the skin, each lasting about 2 hours. Once patients tolerate the infusions with minimal or no side effects, they may be given the remaining infusions on an outpatient basis. Patients who relapse after their initial response to alemtuzumab are given cyclosporine to see if this drug will boost their immune response.
-Patients receive transfusions, growth factors, and antibiotic therapy, as needed.
-Infection therapy: Patients are given aerosolized pentamidine to protect against lung infections and valacyclovir to protect against herpes infections.
-A blood test is done and vital signs are measured every day while patients receive alemtuzumab.
-Patients have an echocardiogram and 24-hour Holter monitor after the last dose of alemtuzumab.
-Blood tests are done weekly for the first 3 months after alemtuzumab administration, then every other week until 6 months.
Patients return to the NIH for follow-up visits 1 month, 3 months, 6 months, and yearly for 5 years after the last dose of alemtuzumab for the following tests and evaluations:
-Blood test
-Repeat echocardiogram at 3-month visit
-Repeat bone marrow biopsy 6 months and 12 months after alemtuzumab, then as clinically indicated for 5 years.
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INCLUSION CRITERIA: Relapsed severe aplastic anemia after initial hematologic response to a prior course of h-ATG or r-ATG based immunosuppression Or Refractory severe aplastic anemia not responding to both horse-ATG and rabbit ATG-based immunosuppression The criteria for severe aplastic anemia are two of the three criteria: -Absolute neutrophil count less than or equal to 500 /mm(3) -Platelets to less than or equal to 20,000/mm(3) -Absolute reticulocyte count less than 60,000 /microL Age greater than or equal to 2 years old and greater than 12 kg Prospective subjects or their parent(s)/responsible guardian(s) must be able to comprehend and be willing to sign an informed consent. EXCLUSION CRITERIA: Known Diagnosis of Fanconi's anemia Evidence of a clonal disorder on cytogenetics. In the refractory disease setting, prospective subjects with super severe neutropenia (ANC less than 200 /microL) will not be excluded if results of cytogenetics are not available or pending. Infection not adequately responding to appropriate therapy HIV positivity Failure to discontinue the herbal supplements Echinacea purpurea or Usnea barbata (Old Man's Beard) within 2 weeks of enrollment 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 Previous hypersensitivity to alemtuzumab or its components Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible Current pregnancy, or unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential Not able to understand the investigational nature of the study or give informed consent
Relapsed severe aplastic anemia after initial hematologic response to a prior course of h-ATG or r-ATG based immunosuppression
Or
Refractory severe aplastic anemia not responding to both horse-ATG and rabbit ATG-based immunosuppression
The criteria for severe aplastic anemia are two of the three criteria:
-Absolute neutrophil count less than or equal to 500 /mm(3)
-Platelets to less than or equal to 20,000/mm(3)
-Absolute reticulocyte count less than 60,000 /microL
Age greater than or equal to 2 years old and greater than 12 kg
Prospective subjects or their parent(s)/responsible guardian(s) must be able to comprehend and be willing to sign an informed consent.
EXCLUSION CRITERIA:
Known Diagnosis of Fanconi's anemia
Evidence of a clonal disorder on cytogenetics. In the refractory disease setting, prospective subjects with super severe neutropenia (ANC less than 200 /microL) will not be excluded if results of cytogenetics are not available or pending.
Infection not adequately responding to appropriate therapy
HIV positivity
Failure to discontinue the herbal supplements Echinacea purpurea or Usnea barbata (Old Man's Beard) within 2 weeks of enrollment
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
Previous hypersensitivity to alemtuzumab or its components
Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
Current pregnancy, or unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
Not able to understand the investigational nature of the study or give informed consent
Principal Investigator
Referral Contact
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