Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

• Status: Completed
• Trial end date: 2010-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety of the drug oxandrolone (a type of androgen steroid) in patients with Fanconi anemia (FA), and to determine if this drug can help in the treatment of bone marrow failure in these patients. Androgen steroids are male hormones that can stimulate the production of red blood cells (the cells which carry oxygen in the blood) and platelets (cells that help blood clot).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborator: FDA Office of Orphan Products Development
• Treatments: Oxandrolone

Criteria

Inclusion Criteria:

1. Patients must be diagnosed with Fanconi anemia that is documented by a positive test for increased chromosomal breakage with mitomycin C or diepoxybutane.

2. 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

3. Negative pregnancy test by hCG testing, if of child-bearing potential.

4. Agreement to use a medically approved form of birth control, if of child-bearing potential.

5. Signed informed consent by the patient or legally authorized representative.

6. Patients must be 14 kg.

7. 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:

1. Malignancy

2. Concurrent enrollment in any other study using an investigational drug.

3. Concurrent use of anticoagulants.

4. Use of androgen therapy within last three months.

5. 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.

6. Patients with renal disease as defined by serum creatinine greater than or equal to 1.5 x the upper limit of normal for age.

7. Patients less than 14 kg.

8. Patients who have failed previous therapy with oxymetholone.

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