Status:
COMPLETED
Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
FDA Office of Orphan Products Development
Conditions:
Fanconi Anemia
Eligibility:
All Genders
Phase:
PHASE1
Brief 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 o...
Detailed Description
The primary purpose of this study is to evaluate the safety of the drug oxandrolone in patients with Fanconi anemia (FA), and secondarily to determine if this drug can help in the treatment of bone ma...
Eligibility Criteria
Inclusion
- Patients must be diagnosed with Fanconi anemia that is documented by a positive test for increased chromosomal breakage with mitomycin C or diepoxybutane.
- 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
- Negative pregnancy test by hCG testing, if of child-bearing potential.
- Agreement to use a medically approved form of birth control, if of child-bearing potential.
- Signed informed consent by the patient or legally authorized representative.
- Patients must be 14 kg.
- 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
- Malignancy
- Concurrent enrollment in any other study using an investigational drug.
- Concurrent use of anticoagulants.
- Use of androgen therapy within last three months.
- 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.
- Patients with renal disease as defined by serum creatinine greater than or equal to 1.5 x the upper limit of normal for age.
- Patients less than 14 kg.
- Patients who have failed previous therapy with oxymetholone.
- \-
Key Trial Info
Start Date :
July 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2010
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT00243399
Start Date
July 1 2004
End Date
January 1 2010
Last Update
July 15 2011
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039