Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita (NCT01001598) | Clinical Trial Compass
TerminatedPhase 1/2
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Stopped: Study was terminated due to under enrollment
United States5 participantsStarted 2009-11
Plain-language 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 masculinizing side effects, indicating a need for a different medication. Danazol is a less potent androgen,and may therefore have fewer masculinizing side effects. Danazol is currently approved by the Food and Drug Administration (FDA) for the treatment of other diseases, but it has never been studied in patients with FA and DC.
The main purpose of this study is to see if danazol is a safe treatment for FA and DC. Specifically,we would like to determine:
* the best dose of danazol;
* how fast hemoglobin (a protein that carries oxygen in the blood) levels rise in FA and DC patients receiving danazol therapy; and
* the genetic pattern (known as expression profile) of certain cells in response to danazol, which can predict how well people respond to the medication.
Subjects who enroll in the study will be treated with danazol for up to 24 weeks (about 6 months), and will have up to 11 study visits, including followup visits at 38 weeks (9 months) and 52 weeks (one year).
Who can participate
Age range3 Years
SexALL
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Inclusion criteria
✓. 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 criteria
✕. Malignancy
✕. Concurrent enrollment in any other study using an investigational drug.
What they're measuring
1
Number of Participants With Toxicity Associated With Danazol Therapy: Virilization, and/or New or Progressive Evidence of Either Hepatic or Renal Toxicity at a Grade II Level Using National Cancer Institute Common Toxicity Criteria (NCI-CTC).