NCT00243399

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jul 2004

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2004

Completed
1.3 years until next milestone

First Submitted

Initial submission to the registry

October 20, 2005

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 24, 2005

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2009

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2010

Completed
Last Updated

July 15, 2011

Status Verified

July 1, 2011

Enrollment Period

5.4 years

First QC Date

October 20, 2005

Last Update Submit

July 14, 2011

Conditions

Fanconi Anemia

Keywords

Fanconi anemiaFA

Outcome Measures

Primary Outcomes (1)

  • Toxicity associated with oxandrolone therapy in patients with Fanconi Anemia

    One year

Secondary Outcomes (1)

  • Hematologic response rate in Fanconi Anemia patients receiving oxandrolone therapy

    One year

Study Arms (1)

Oxandrolone

EXPERIMENTAL
Drug: Oxandrolone

Interventions

OxandroloneDRUG

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.

Also known as: Oxandrin
Oxandrolone

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

MeSH Terms

Conditions

Fanconi Anemia

Interventions

Oxandrolone

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

AndrostanolsAndrostanesSteroidsFused-Ring CompoundsPolycyclic Compounds

Study Officials

  • Franklin O Smith, M.D.

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

October 20, 2005

First Posted

October 24, 2005

Study Start

July 1, 2004

Primary Completion

December 1, 2009

Study Completion

January 1, 2010

Last Updated

July 15, 2011

Record last verified: 2011-07

Locations

US(1)