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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01001598 |
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Date of registration:
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22/10/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
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Scientific title:
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Phase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita |
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Date of first enrolment:
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November 2009 |
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Target sample size:
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5 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01001598 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Colin A Sieff, MB.BCh |
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Address:
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Telephone:
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Email:
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Affiliation:
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Boston Children's Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. 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).
2. 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
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 either 3 years of age or > 14 kg.
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 liver disease as defined by SGOT, SGPT or bilirubin greater than the
upper limit of normal.
6. Patients with renal disease as defined by serum creatinine greater than the upper
limit of normal for age.
7. Patients less than either 3 years of age or 14 kg.
8. Patients who have HLA matched sibling donors.
Age minimum:
3 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fanconi Anemia
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Dyskeratosis Congenita
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Intervention(s)
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Drug: danazol
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Primary Outcome(s)
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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).
[Time Frame: 48 weeks (24 weeks treatment and 24 weeks extension phase)]
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Secondary Outcome(s)
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The Gene Expression Profile of Progenitor Cells in Response to Danazol, Both to Predict Responsiveness and to Screen for Small Molecules That Show a Profile Similar to That of Responsive Patients
[Time Frame: Baseline and 24 weeks]
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The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy
[Time Frame: 12, 18 and 24 weeks]
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Secondary ID(s)
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09-03-0131
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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