Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia

NCT00005935 | Completed Phase 2

• 2 other identifiers: 00015700-H-0157
• Study Type: interventional
• Target: 130
• Locations: 1 country
• Sites: 1

Brief Summary

This study will examine the safety and effectiveness of a new drug combination for treating patients with severe aplastic anemia. Patients with aplastic anemia produce too few blood cells, causing fatigue, easy bruising and bleeding, and susceptibility to infections. In many cases, the very low blood counts result from an autoimmune process-that is, the patient's own immune system suppresses production of blood cells by the bone marrow. Although immune-suppressing drugs, such as cyclosporine, can restore normal cell counts, many patients have disease relapses. These patients require long-term therapy with cyclosporine, which can cause harmful side effects. This study will examine whether a lower dose of cyclosporine given together with mycophenolate mofetil (MMF) can maintain blood counts as effectively as full-dose cyclosporine treatment, and whether MMF alone can reduce the chances of future relapses. Patients 4 years of age and older with severe aplastic anemia who have relapsed after immune suppressing therapy may be eligible for this study. Participants will be randomly assigned to receive either standard cyclosporine therapy or experimental therapy with cyclosporine and MMF. Patients receiving standard cyclosporine therapy will receive a full dose of the drug for at least 3 months. Those taking both cyclosporine and MMF will take MMF plus half-dose cyclosporine for 3 months and continue MMF for an additional 6 months. Both drugs are taken twice a day by mouth. All patients will have about 120 milliliters (4 ounces) of blood drawn at the beginning of the study to evaluate immune system activity and bone marrow function, and to look for genetic material of certain viruses. Bone marrow aspirations and biopsies will be done at the beginning of the study, and at 6 and 12 months. For these tests, the area of the hip is anesthetized and a special needle is used to draw bone marrow from the hipbone. The patient's local doctor will be asked to do blood tests for chemistries, liver function and cyclosporine levels weekly for the first month and then every other week. Patients will return to NIH for evaluations 3, 6 and 12 months after treatment and then once a year. About 100 ml (7 tablespoons) of blood will be drawn at each visit.

Conditions

Aplastic Anemia

Keywords

Immunosuppression; T Cells; Autoimmunity; Bone Marrow Failure; Hematopoiesis; Relapsing Aplastic Anemia

Interventions

Mycophenolate mofetil DRUG

Cyclosporine DRUG

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

Subjects with a history of severe aplastic anemia successfully treated by immunosuppression will be included. Subjects with relapse, as defined above by either return of blood counts to satisfy criteria for severity or consistently declining blood counts will be included. Subjects age 4 and above will be included. Subjects with the presence of a medical or surgical condition making survival for at least 3 months unlikely will be excluded. Subjects with inability to confer informed consent or assent, in the case of a child, either written or verbal, will be excluded.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Heart, Lung and Blood Institute (NHLBI)

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Young NS, Barrett AJ. The treatment of severe acquired aplastic anemia. Blood. 1995 Jun 15;85(12):3367-77. No abstract available.

  PMID: 7780125 BACKGROUND

• Young NS. Autoimmunity and its treatment in aplastic anemia. Ann Intern Med. 1997 Jan 15;126(2):166-8. doi: 10.7326/0003-4819-126-2-199701150-00014. No abstract available.

  PMID: 9005753 BACKGROUND

• Rosenfeld SJ, Kimball J, Vining D, Young NS. Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood. 1995 Jun 1;85(11):3058-65.

  PMID: 7756640 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic; Autoimmune Diseases; Bone Marrow Failure Disorders

Interventions

Mycophenolic Acid; Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 2
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: July 6, 2000
• First Posted: July 7, 2000
• Study Start: June 1, 2000
• Study Completion: March 1, 2002
• Last Updated: March 4, 2008

Record last verified: 2002-03

Locations

US (1)