search
Back to results

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

Primary Purpose

Fanconi Anemia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Oxandrolone
Sponsored by
Children's Hospital Medical Center, Cincinnati
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fanconi Anemia focused on measuring Fanconi anemia, FA

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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 Criteria: 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. -

Sites / Locations

  • Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Oxandrolone

Arm Description

Outcomes

Primary Outcome Measures

Toxicity associated with oxandrolone therapy in patients with Fanconi Anemia

Secondary Outcome Measures

Hematologic response rate in Fanconi Anemia patients receiving oxandrolone therapy

Full Information

First Posted
October 20, 2005
Last Updated
July 14, 2011
Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
FDA Office of Orphan Products Development
search

1. Study Identification

Unique Protocol Identification Number
NCT00243399
Brief Title
Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Official Title
A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
July 2011
Overall Recruitment Status
Completed
Study Start Date
July 2004 (undefined)
Primary Completion Date
December 2009 (Actual)
Study Completion Date
January 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
FDA Office of Orphan Products Development

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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 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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi Anemia
Keywords
Fanconi anemia, FA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Oxandrolone
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Oxandrolone
Other Intervention Name(s)
Oxandrin
Intervention Description
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.
Primary Outcome Measure Information:
Title
Toxicity associated with oxandrolone therapy in patients with Fanconi Anemia
Time Frame
One year
Secondary Outcome Measure Information:
Title
Hematologic response rate in Fanconi Anemia patients receiving oxandrolone therapy
Time Frame
One year

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Criteria: 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. -
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Franklin O Smith, M.D.
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

We'll reach out to this number within 24 hrs