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Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

NCT00243399 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2011-07-15

No results posted yet for this study

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).

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.

Sponsors & Collaborators

  • FDA Office of Orphan Products Development

    collaborator FED

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Franklin O Smith, M.D. · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-07-31
Primary Completion
2009-12-31
Completion
2010-01-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00243399 on ClinicalTrials.gov