Medical Treatment for Diamond Blackfan Anemia
|First Received Date ICMJE||November 3, 1999|
|Last Updated Date||March 3, 2008|
|Start Date ICMJE||July 1998|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE||Not Provided|
|Original Primary Outcome Measures ICMJE||Not Provided|
|Change History||Complete list of historical versions of study NCT00001749 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE||Not Provided|
|Original Secondary Outcome Measures ICMJE||Not Provided|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Medical Treatment for Diamond Blackfan Anemia|
|Official Title ICMJE||Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A|
Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood cells and platelets, are normal.
Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure, relapse, side-effects, increased morbidity, and even death. Two drugs, antithymocyte globulin (ATG) and cyclosporin have been used to treat DBA, but have only provided occasional responses. No study has ever combined these two drugs for the treatment of DBA.
This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA.
Diamond Blackfan anemia (DBA) is a constitutional pure red cell aplasia of unknown etiology. There is laboratory evidence for an immune mechanism and most patients respond to corticosteroids. However the relapse and failure rate are high, and corticosteroids are associated with many short and long term side effects. Patients who do not respond or who do not tolerate corticosteriods require lifelong red blood cell transfusion and iron chelation therapy. Allogeneic bone marrow transplantation is an option for those with a related histocompatible donor, but this procedure is associated with high mortality and morbidity. Other therapies have been tried without general success. Occasional responses to either ATG or cyclosporine have been reported, but no study has used both ATG and cyclosporine. In other blood/bone marrow disorders of immune etiology these drugs have synergistic effects. We propose a Phase II study to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of Diamond Blackfan anemia.
|Study Type ICMJE||Interventional|
|Study Phase||Phase 2|
|Study Design ICMJE||Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment
|Study Arm (s)||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Completed|
|Completion Date||July 2005|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
Diagnosis of DBA as characterized by a hyporegenerative anemia presenting in early childhood with reticulocytopenia, and low or absent erythroid precursors in the bone marrow.
Transfusion-dependence due to steroid failure or intolerance of steroid side effects.
Ineligible for or declining an allogeneic transplant.
Ages 3 to 75.
Serum creatinine greater than 2 times normal or a creatinine clearance less than 50% normal.
SGPT or SGOT greater than 5 times normal.
History of epilepsy (any seizures besides childhood febrile seizures).
Current pregnancy or unwillingness to take oral contraceptives if menstruating.
Positive diepoxybutane (DEB) test for Fanconi anemia.
Inability or unwillingness to sign an informed consent, either by the patient, or in the case of a minor, by the parent or guardian responsible for the patient.
Underlying organ failure and/or those with a Karnofsky performance status of less than 1.
Treatment with androgens, prednisone greater than 10 mg/day, growth factors, or other immunosuppressive therapies within one month of protocol entry.
Ongoing treatment with Beta-adrenergic blocking drugs.
Previous treatment with ATG and concurrent CSA. Previous treatment with either drug alone is acceptable if greater than one year prior to study entry.
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Location Countries ICMJE||United States|
|NCT Number ICMJE||NCT00001749|
|Other Study ID Numbers ICMJE||980144, 98-H-0144|
|Has Data Monitoring Committee||Not Provided|
|Responsible Party||Not Provided|
|Study Sponsor ICMJE||National Heart, Lung, and Blood Institute (NHLBI)|
|Collaborators ICMJE||Not Provided|
|Investigators ICMJE||Not Provided|
|Information Provided By||National Institutes of Health Clinical Center (CC)|
|Verification Date||July 2005|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP