Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Fanconi Anemia
About this trial
This is an interventional treatment trial for Fanconi Anemia focused on measuring Fanconi anemia, FA
Eligibility Criteria
Inclusion Criteria: Patients must be diagnosed with Fanconi anemia that is documented by a positive test for increased chromosomal breakage with mitomycin C or diepoxybutane. At least the following peripheral blood cytopenias: (without transfusion) Absolute neutrophil count < 500/mm3 or Platelet count < 30,000/mm3 or Hemoglobin < 8.0 gm/dl Negative pregnancy test by hCG testing, if of child-bearing potential. Agreement to use a medically approved form of birth control, if of child-bearing potential. Signed informed consent by the patient or legally authorized representative. Patients must be 14 kg. Male patients will be included until the time of puberty. With the onset of puberty, they will be included until the testosterone levels reach 100 ng/dl at which time they will be excluded from the study. Exclusion Criteria: Malignancy Concurrent enrollment in any other study using an investigational drug. Concurrent use of anticoagulants. Use of androgen therapy within last three months. Patients with severe liver disease as defined by SGOT or SGPT greater than or equal to 2.5x the upper limit of normal or total bilirubin greater than or equal to 1.5x the upper limit of normal. Patients with renal disease as defined by serum creatinine greater than or equal to 1.5 x the upper limit of normal for age. Patients less than 14 kg. Patients who have failed previous therapy with oxymetholone. -
Sites / Locations
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Experimental
Oxandrolone