Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-26 @ 12:19 PM
Ignite Modification Date:
2025-12-26 @ 12:19 PM
NCT ID:
NCT03312400
Eligibility Criteria:
* INCLUSION CRITERIA:
1. Age-adjusted telomere length less than or equal to the first percentile by flow-FISH method. In patients with a known pathogenic or likely pathogenic mutation in a telomere maintenance gene, age adjusted telomere length less than or equal to the 10th percentile is sufficient.
2. A mutation in telomere maintenance genes (TERT, TERC, DKC1, TINF2, NHP2, NOP10, WRAP53, TERF2, PARN, RTEL1, ACD, CTC1, USB1) as tested in a CLIA (or international equivalent) certified laboratory
3. Age greater than or equal to 3 years
4. Weight greater than or equal to 12 Kg
AND
5. At least one of the following criteria:
1. Anemia with a hemoglobin less than or equal to 10 g/dL without red blood cell transfusion
2. Thrombocytopenia with a platelet count less than or equal to 50,000/microliter without transfusion
3. Neutropenia with an absolute neutrophil count less than or equal to 1,000/ microliter
OR
Pulmonary fibrosis diagnosed by either a lung biopsy or computed tomography scan of the chest according to guidelines from the American Thoracic Society and European Respiratory Society.
OR
6. Hepatic fibrosis diagnosed by Transient Elastography by Fibroscan value greater than 10 kpa or US evidence of cirrhotic liver or splenomegaly, or transjugular liver biopsy demonstrating fibrosis.
EXCLUSION CRITERIA:
1. Patients on androgen hormones to include testosterone or high dose estrogen (estradiol 0.5 mg/day or greater) for the12 months prior to enrollment
2. Patients with active thrombosis or thromboembolic disease and history of such events, undiagnosed abnormal genital bleeding, porphyria, androgendependent tumor, or prostatic hypertrophy
3. Patients with pulmonary fibrosis who are receiving anti-fibrotic drug treatment, such as pirfenidone or nintedanib unless stable on anti-fibrotic drug for at least 6 months prior to starting on danazol as demonstrated by PFTs.
4. Patients with active hepatitis B or C
5. Patients who have received a bone marrow transplant
6. Patient with other hereditary bone marrow failure syndromes such as Fanconi anemia or Diamond Blackfan anemia
7. Patients with infections not adequately responding to appropriate therapy
8. Current pregnancy, or unwillingness to take oral contraceptives or use the barrier methods of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of the study
9. Lactating women, due to the potentially harmful effects on the nursing child
10. Patients with cancer who are actively receiving systemic chemotherapeutic treatment or who take drugs with hematological effects
11. Patients with decompensated liver disease to include persistent ascites, encephalopathy, variceal hemorrhage, or MELD score of 10 or greater
12. Inability to understand the investigational nature of the study or to give informed consent or without a legally authorized representative or surrogate that can provide informed consent
13. Inability to swallow a capsule
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
3 Years
Maximum Age:
99 Years
Study:
NCT03312400
Study Brief:
Protocol Section:
NCT03312400