Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

PHASE1CompletedINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

July 31, 2004

Primary Completion Date

December 31, 2009

Study Completion Date

January 31, 2010

Conditions
Fanconi Anemia
Interventions
DRUG

Oxandrolone

Subjects will be started on a dose of oxandrolone 0.04 mg/kg/day orally, once/day. After 16 weeks, patients will be assessed for hematologic improvement. If this criteria is not met after 16 weeks, the dose will be escalated to 0.08 mg/kg/day. If no improvement is noted after 16 weeks on the escalated dose, oxandrolone therapy will be discontinued.

Trial Locations (1)

45229-3039

Cincinnati Children's Hospital Medical Center, Cincinnati

All Listed Sponsors
collaborator

FDA Office of Orphan Products Development

FED

lead

Children's Hospital Medical Center, Cincinnati

OTHER

NCT00243399 - Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia | Biotech Hunter | Biotech Hunter