drugset / Trial / NCT00243399
Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Phase 1
Completed
10 enrolled
Children's Hospital Medical Center, Cincinnati
FDA Office of Orphan Products Development · collab
Non-randomizedSingle-groupOpen-labelTreatment
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).
Timeline
- Start
- 2004-07
- Primary completion
- 2009-12
- Completion
- 2010-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Oxandrolone | Small molecule | 0.04 mg/kg | Oral |
| Subject | Oxandrolone | Small molecule | 0.08 mg/kg | Oral |