Comparing Therapies for the Treatment of Severe Aplastic Anemia

Completed · Phase 2

Conditions studied: Severe Aplastic Anemia (SAA)

In brief

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.

Key facts

Study ID

NCT00001626

Run by

National Heart, Lung, and Blood Institute (NHLBI)

People needed

33

Starts

1997-06-02

Expected to finish

2008-03-03

Last updated by the study team

2020-09-22

Who can join

Age: 15 and older, up to 110. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• 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.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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