Status:
TERMINATED
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Lead Sponsor:
Boston Children's Hospital
Conditions:
Fanconi Anemia
Dyskeratosis Congenita
Eligibility:
All Genders
3+ years
Phase:
PHASE1
PHASE2
Brief Summary
Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculi...
Detailed Description
Eligible patients with either Fanconi anemia (FA) or Dyskeratosis congenita (DC) will initially receive danazol at a dose of 5 mg/kg/d orally, rounded to the nearest 100 mg. For the first 8 weeks, the...
Eligibility Criteria
Inclusion
- Patients must be diagnosed with FA that is documented by a positive test for increased chromosomal breakage with mitomycin C or diepoxybutane. DC patients must have clinical features consistent with the diagnosis, abnormally short lymphocyte telomeres \< 1st centile by flow-FISH evaluation, or mutation in one of the known DC genes (DKC1, TERT, TERC, TINF2, NOP10, NHP2).
- At least the following peripheral blood cytopenias: (without transfusion) Absolute neutrophil count \< 500/uL or Platelet count \< 30,000/uL 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 either 3 years of age or \> 14 kg.
Exclusion
- 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 liver disease as defined by SGOT, SGPT or bilirubin greater than the upper limit of normal.
- Patients with renal disease as defined by serum creatinine greater than the upper limit of normal for age.
- Patients less than either 3 years of age or 14 kg.
- Patients who have HLA matched sibling donors.
Key Trial Info
Start Date :
November 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2014
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT01001598
Start Date
November 1 2009
End Date
May 1 2014
Last Update
February 19 2019
Active Locations (1)
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1
Children's Hospital Boston
Boston, Massachusetts, United States, 02115