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Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

This study has been completed.
Sponsor:
Collaborator:
FDA Office of Orphan Products Development
Information provided by:
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT00243399
First received: October 20, 2005
Last updated: July 14, 2011
Last verified: July 2011
History of Changes
  Purpose

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 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia

Resource links provided by NLM:

Drug Information available for: Oxandrolone
U.S. FDA Resources

Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • Toxicity associated with oxandrolone therapy in patients with Fanconi Anemia [ Time Frame: One year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Hematologic response rate in Fanconi Anemia patients receiving oxandrolone therapy [ Time Frame: One year ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: July 2004
Study Completion Date: January 2010
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Oxandrolone 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.
Other Name: Oxandrin

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 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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

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  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00243399

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
FDA Office of Orphan Products Development
Investigators
Principal Investigator: Franklin O Smith, M.D. Children's Hospital Medical Center, Cincinnati
  More Information

No publications provided

Responsible Party: Franklin O. Smith, Cincinnati Children's Hospital Medical Center
ClinicalTrials.gov Identifier: NCT00243399     History of Changes
Other Study ID Numbers: 2539, FD-R-002539-01
Study First Received: October 20, 2005
Last Updated: July 14, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fanconi anemia
FA

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Pancytopenia
Fanconi Anemia
Fanconi Syndrome
Hematologic Diseases
Bone Marrow Diseases
Anemia, Hypoplastic, Congenital
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
 Kidney Diseases
Urologic Diseases
Renal Tubular Transport, Inborn Errors
Metabolism, Inborn Errors
Oxandrolone
Anabolic Agents
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions
Androgens

ClinicalTrials.gov processed this record on May 22, 2013