Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00243399
• Date of registration: 20/10/2005
• Prospective Registration: No
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
• Scientific title: A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia
• Date of first enrolment: July 2004
• Target sample size: 10
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00243399
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Franklin O Smith, M.D.
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patients must be diagnosed with Fanconi anemia that is documented by a positive test
for increased chromosomal breakage with mitomycin C or diepoxybutane.

2. 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

3. Negative pregnancy test by hCG testing, if of child-bearing potential.

4. Agreement to use a medically approved form of birth control, if of child-bearing
potential.

5. Signed informed consent by the patient or legally authorized representative.

6. Patients must be 14 kg.

7. 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 Criteria:

1. Malignancy

2. Concurrent enrollment in any other study using an investigational drug.

3. Concurrent use of anticoagulants.

4. Use of androgen therapy within last three months.

5. 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.

6. Patients with renal disease as defined by serum creatinine greater than or equal to
1.5 x the upper limit of normal for age.

7. Patients less than 14 kg.

8. Patients who have failed previous therapy with oxymetholone.

-



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Fanconi Anemia

Intervention(s)

Drug: Oxandrolone

Primary Outcome(s)

Toxicity associated with oxandrolone therapy in patients with Fanconi Anemia [Time Frame: One year]

Secondary Outcome(s)

Hematologic response rate in Fanconi Anemia patients receiving oxandrolone therapy [Time Frame: One year]

Secondary ID(s)

2539

FD-R-002539-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

FDA Office of Orphan Products Development