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Sponsored by: |
FDA Office of Orphan Products Development |
Information provided by: | FDA Office of Orphan Products Development |
ClinicalTrials.gov Identifier: | NCT00243399 |
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).
Condition | Intervention | Phase |
Fanconi Anemia |
Drug: Oxandrolone |
Phase I |
MedlinePlus related topics: | Anemia |
ChemIDplus related topics: | Oxandrolone |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia |
Estimated Enrollment: | 10 |
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 marrow failure in these patients. It is hoped that oxandrolone will have less side effects than oxymetholone, the androgen used most frequently in the short-term treatment of bone marrow failure in FA patients. Subjects will be enrolled for approximately 18 to 30 months (12 - 24 months of treatment and 6 months additional monitoring). The oxandrolone starting dose is 0.04mg/kg/day. Study monitoring includes weekly complete blood counts, monthly serum chemistry labs, quarterly physical examinations including virilization exams and liver ultrasounds. Semi-annually, hand radiographs are obtained for bone maturation and behavioral assessments are conducted to detect any aggressive behavior or mood changes. If no improvement n the subject’s blood counts are noted after 4 months of therapy, the dose will be increased to 0.08mg/kg/day for a period of 4 more months. If no improvement is noted after a total of eight months, oxandrolone will be discontinued. If the blood counts show improvement, then the drug will continue for a minimum of twelve months. Subjects may remain on study and receive a total of 24 months of therapy if they have a response in their blood counts without unacceptable side effects. Post-treatment monitoring includes blood work and ultrasound every three months, and hand radiograph at six months.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Patients who have failed previous therapy with oxymetholone.
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Contact: Moira Haren, M.S., PA-C | 513-636-9972 | moira.haren@cchmc.org |
Contact: Robin Mueller, RN | 513-636-3218 |
United States, Ohio | |||||
Cincinnati Children's Hospital Medical Center | Recruiting | ||||
Cincinnati, Ohio, United States, 45229-3039 | |||||
Principal Investigator: Franklin O Smith, M.D. |
Principal Investigator: | Franklin O Smith, M.D. | Children's Hospital Medical Center, Cincinnati |
Study ID Numbers: | 2539, FD-R-002539-01 |
First Received: | October 20, 2005 |
Last Updated: | October 26, 2005 |
ClinicalTrials.gov Identifier: | NCT00243399 |
Health Authority: | United States: Food and Drug Administration |
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