Trial Information

Summary: A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

What is the purpose of this study?

The purpose of this research study is to evaluate the safety of the drug oxandrolone (a type of androgen steroid) to determine if this drug can help in the treatment of bone marrow failure in patients with Fanconi anemia.

Who can participate?

The study is open to patients with Fanconi anemia who show signs of bone marrow failure. The absolute neutrophil (granulocyte) count must be lower than 500/mm3 or the platelet count must be lower than 30,000/mm3 or the hemoglobin must be lower than 8.0 gm/dl. Patients must weigh more than 31 pounds. Females must have a negative pregnancy test. Males will be eligible until the time of puberty; at that time the testosterone level will be checked and must be below 100 ng/dl.

What is involved?

If accepted for enrollment in this study, a history, physical exam, behavioral assessment, laboratory tests, hand x-ray, and liver ultrasound is required. These tests and exams are repeated throughout the study. Weekly complete blood counts are required. Patients will receive the study drug oxandrolone (which is a pill taken by mouth) for twelve months and monitored for an additional six months.

What are the benefits?

There may not be a direct medical benefit for you if you participate in this study. Potential benefits may be improved blood counts, although this drug is not a cure.

What are the risks?

The most serious risk associated with androgen steroid therapy is the development of cysts in the liver. Increased masculine (male-like) features in both boys and girls may occur. Additional side effects include water retention, decreased tolerance to sugar, weight gain, increased blood pressure, behavioral changes, nausea, vomiting, diarrhea, and changes in liver function tests. The physical exams will include an examination of testicles in males and the clitoris in females.

Will I be paid for participating in this study?

No. All care that is provided at Cincinnati Children's Hospital Medical Center General Clinical Research Center in Cincinnati, Ohio will be paid for by research funds. Any care not provided at this facility may be your or your insurance carriers’ responsibility.

If you would like to learn more about participating in this study, please send an e-mail message using the form below.