|
Comparing Therapies for the Treatment of Severe Aplastic Anemia Clinical Trials Information presented on Clinical Trials Search isn't designed to be a substitute for certified healthcare advice, travels to or professional assistance using a genuine medical doctor. We are not physicians. Always confer with your dr. about Comparing Therapies for the Treatment of Severe Aplastic Anemia conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Comparing Therapies for the Treatment of Severe Aplastic Anemia Clinical research trials and Comparing Therapies for the Treatment of Severe Aplastic Anemia medical trials happen in hundreds of places across the United States. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually measure the effectualness of new drugs. The intention of the studies / undertakings is to solve certain human healthcare questions. Clinical trials are a popular manner for mDs, government agencies, and private sector companies to locate treatments for all forms of circumstances, such as Comparing Therapies for the Treatment of Severe Aplastic Anemia. Comparing Therapies for the Treatment of Severe Aplastic Anemia Clinical Trials and other clinical trials allow for volunteers to undergo medical treatment choices before they are available to the general public. Some times the human subjects get treatment for free of charge, and sometimes they are paid for their time. Occasionally there is a cost for a Comparing Therapies for the Treatment of Severe Aplastic Anemia clinical trial. Participants frequently get the best healthcare available for their Comparing Therapies for the Treatment of Severe Aplastic Anemia condition. Risks are a reality, nonetheless, and can include extra or frequent physician trips, medical risks (possibly life-jeopardising), and/or the treatment being ineffective. Trials are federally governed with exacting guidelines to protect clinical trials subjects.
|
|
|
|
|
|
|
Home > "C" Clinical Trials Conditions > Comparing Therapies for the Treatment of Severe Aplastic Anemia  Comparing Therapies for the Treatment of Severe Aplastic Anemia
Comparing Therapies for the Treatment of Severe Aplastic Anemia
For Condition: Aplastic Anemia,Hematologic Disease
Status: No longer recruiting
Sponsor(s): National Heart, Lung, and Blood Institute (NHLBI) ,
Synopsis: Severe Aplastic Anemia (SAA) is a rare and very serious blood disorder in which the bone marrow stops producing the cells which make up blood; red blood cells, white blood cells, and platelets. Researchers believe this is caused by an autoimmune reaction, a condition in which the natural defense system of the body begins attacking itself. In SAA the immune system begins attacking the bone marrow. Red blood cells are responsible for carrying oxygen to all of the organ systems in the body, and low numbers (anemia) can cause difficulty breathing and fatigue. Platelets are responsible for normal blood clotting and low numbers can result in easy bruising and bleeding which can be deadly. White blood cells are responsible for fighting infections, and low numbers of these can lead to frequent infections, the most common cause of death in patients with aplastic anemia. SAA can be treated by bone marrow transplant (BMT) or by drugs designed to slow down the immune system (immunosuppressants). BMT can be successful, but it requires a donor with matched bone marrow, making this therapy available only to a few patients. BMT with unmatched bone marrow can fail and cause dangerous side effects. Presently, the two drugs used to treat SAA by slowing down the immune system (immunosuppression) are antithymocyte globulin (ATG) and cyclosporin A (CSA). When used in combination these two drugs can improve most patients' condition. However, one third of the patients who respond to this therapy experience a relapse of SAA. In addition, some patients treated with ATG/CSA can later develop other disorders of the blood. Recently, researchers have found that another immunosuppressive drug called cyclophosphamide, has been successful at treating patients with SAA. In addition, patients treated with cyclophosphamide do not experience relapses or develop other disorders of the blood. In this study researchers would like to compare the combinations of antithymocyte globulin (ATG) and cyclosporin A (CSA) to cyclophosphamide and cyclosporin A (CSA) for the treatment of SAA.
Details: Severe aplastic anemia (SAA) is a disorder with a poor prognosis if untreated. Current accepted therapeutic strategies include bone marrow transplantation (BMT) and immunosuppression, both offering cure or amelioration in the majority of patients. Although BMT is successful using human leukocyte antigen (HLA) matched sibling bone marrow, the 25% probability of finding an HLA identical sibling within a family renders this approach available to only a minority of patients. BMT utilizing HLA-matched, unrelated donors carries a high risk of treatment failure along with considerable toxicity. While combined immunosuppression with both antithymocyte globulin (ATG) and cyclosporine A (CSA) produces hematologic improvement in most patients, relapse is common, occurring in about a third of responders. Late evolution of aplastic anemia to other serious hematologic disorders is a significant problem following successful treatment with ATG/CSA with paroxysmal nocturnal hemoglobinuria (PNH) occurs in approximately 13%, myelodysplasia in about 10%, and acute leukemia in about 7%. Recently, results of immunosuppression in SAA with another potent immunosuppressive agent, cyclophosphamide, were reported in 10 patients. In this small group, the overall response rate was similar to that seen with ATG/CSA, but relapse and late clonal disease were not seen during a median follow-up of greater than 10 years. In the larger randomized trial proposed here, we will compare sustained hematologic response rates to either conventional immunosuppression with ATG/CSA or high dose cyclophosphamide and CSA. Secondary endpoints include response duration, event free survival, and overall survival.
Eligibility:
Study Type: Interventional, Treatment, Efficacy
Minimum Age/Maximum Age: /
Genders: Both
Protocol Entry Criteria: INCLUSION CRITERIA All patients 15 years old or over with SAA not previously treated with immunosuppression and who lack a suitable sibling matched marrow donor will be considered for enrollment. Severe aplastic anemia confirmed at NIH by: a) Bone marrow cellularity less than thirty percent (excluding lymphocytes). b) At least two of the following: Absolute neutrophil count less that 500/mm(3); Platelet count less than 20,000/mm(3); Reticulocyte count less than 60,000/mm(3). EXCLUSION CRITERIA Serum creatinine greater than to 2.5 mg/dl. Cardiac ejection fraction less than 45% by MUGA. Underlying carcinoma (except local cervical, basal cell, squamous cell or melanoma). Current pregnancy or unwilling to take oral contraceptives. Diagnosis of Fanconi anemia or other congenital bone marrow failure syndromes. Evidence of a clonal disorder on cytogenetics. HIV positivity. Inability to understand the investigational nature of the study. Patients who are moribund or have hepatic, renal, cardiac, metabolic or other concurrent diseases of such severity that death within 7-10 days is likely. Previous treatment with ATG, or cyclophosphamide.
Total Enrollment: 182
Location and Contact Information:
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, Maryland, 20892
United States
Additional Information:
Study ID Numbers: 970117; 97-H-0117
Study Start Date: May 2, 1997
Record last reviewed: April 22, 2004
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00001626
Other Hematologic Disease Studies:
1. Medical Treatment for Diamond Blackfan Anemia
2. Evaluation, Treatment, and Training for Patients with Blood Disorders
3. Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow
4. Comparing Therapies for the Treatment of Severe Aplastic Anemia
5. Collection of Blood and Bone Marrow from Normal Volunteers and Patients for Research Purposes
Related Studies:
Other Hematologic Disease Clinical Trials
Other Maryland Clinical Trials
Other Bethesda Clinical Trials
Comparing Therapies for the Treatment of Severe Aplastic Anemia
|
|
|
|
|
|
|
|
