Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

July 14, 2011 updated by: Children's Hospital Medical Center, Cincinnati

A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Cincinnati Children's Hospital Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

    -

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • Oxandrin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Toxicity associated with oxandrolone therapy in patients with Fanconi Anemia
Time Frame: One year
One year

Secondary Outcome Measures

Outcome Measure
Time Frame
Hematologic response rate in Fanconi Anemia patients receiving oxandrolone therapy
Time Frame: One year
One year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital Medical Center, Cincinnati

Collaborators

FDA Office of Orphan Products Development

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2004

Primary Completion (Actual)

December 1, 2009

Study Completion (Actual)

January 1, 2010

Study Registration Dates

First Submitted

October 20, 2005

First Submitted That Met QC Criteria

October 20, 2005

First Posted (Estimate)

October 24, 2005

Study Record Updates

Last Update Posted (Estimate)

July 15, 2011

Last Update Submitted That Met QC Criteria

July 14, 2011

Last Verified

July 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2539
  • FD-R-002539-01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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