Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Completed
FDA Office of Orphan Products Development
Phase 1
2004-07-01
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).
Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Completed
Children's Hospital Medical Center, Cincinnati
Phase 1
2004-07-01
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).
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Terminated
Boston Children's Hospital
Phase 1/Phase 2
2009-11-01
Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure
syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can
cause unwanted masculinizing side effects, indicating a need for a different medication.
Danazol is a less potent androgen,and may therefore have fewer masculinizing side effects.
Danazol is currently approved by the Food and Drug Administration (FDA) for the treatment of
other diseases, but it has never been studied in patients with FA and DC.
The main purpose of this study is to see if danazol is a safe treatment for FA and DC.
Specifically,we would like to determine:
- the best dose of danazol;
- how fast hemoglobin (a protein that carries oxygen in the blood) levels rise in FA and
DC patients receiving danazol therapy; and
- the genetic pattern (known as expression profile) of certain cells in response to
danazol, which can predict how well people respond to the medication.
Subjects who enroll in the study will be treated with danazol for up to 24 weeks (about 6
months), and will have up to 11 study visits, including followup visits at 38 weeks (9
months) and 52 weeks (one year).
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