Status:
COMPLETED
Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Anemia, Aplastic
Anemia, Hypoplastic
Eligibility:
All Genders
2-82 years
Phase:
PHASE2
Brief Summary
Background: * Severe plastic anemia can lead to problems with bone marrow platelet production and result in low blood platelet counts, which require frequent platelet transfusions to improve blood cl...
Detailed Description
Severe aplastic anemia (SAA), characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) a...
Eligibility Criteria
Inclusion
- All patients 2 years old or over with SAA who have failed initial immunosuppression with r-ATG/CsA and are not candidates for a matched sibling marrow transplantation will be considered for enrollment. Patients who have a suitable matched sibling donor will be referred for consideration of allogeneic bone marrow transplantation. Patients not willing to undergo transplantation will be considered for enrollment. Eligibility will be determined on another screening Hematology Branch protocol (97-H-0041) or another active Hematology branch protocol. The time between determination of eligibility and signing consent to participate on this protocol and initiate treatment on this protocol will not exceed 90 days.
- INCLUSION CRITERIA:
- Diagnosed with SAA characterized by:
- Bone marrow cellularity \< 30% (excluding lymphocytes)
- At least two of the following:
- i. Absolute neutrophil count \<500/ microL
- ii. Platelet count \<20,000/ microL
- iii. Reticulocyte count \<60,000/ microL
- Failure to respond to an initial course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment or a suboptimal response to initial therapy defined by both platelet and reticulocyte count \< 50,000 /microL at 3 months post-treatment
- Age greater than or equal to 2 years of age
- EXCLUSION CRITERIA:
- Diagnosis of Fanconi anemia. Patients with very severe neutropenia (ANC \< 200 /microL) will not be excluded initially if results of Fanconi anemia testing are not available or pending. If evidence of Fanconi anemia is later identified, the subject will go off study.
- Evidence of a clonal disorder on cytogenetics. Patients with very severe neutropenia (ANC \< 200/uL) will not be excluded initially if results of cytogenetics are not available or pending. If evidence of a clonal disorder is later identified, the subject will go off study.
- Patients who received prior course(s) of alemtuzumab will not be excluded.
- Infection not adequately responding to appropriate therapy
- HIV seropositivity
- 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.
- Subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
- Serum creatinine \> 2.5 mg/dL
- Current pregnancy, breast-feeding or unwillingness to refrain from pregnancy if of child bearing potential
- Inability to understand the investigational nature of the study or give informed consent
Exclusion
Key Trial Info
Start Date :
August 31 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2016
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT00944749
Start Date
August 31 2009
End Date
December 1 2016
Last Update
March 23 2021
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