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The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis

Study Purpose

TELO-SCOPE is a national, multi-centre, double-blind, placebo-controlled, randomised (2:1) trial which will test the hypothesis that, compared to placebo, the addition of danazol to standard of care in pulmonary fibrosis associated with short telomeres is safe and will result in reduced telomere attrition.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	5 Years and Over
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Males and females aged >5 years, able to take capsules orally. 2. Fibrosing interstitial pneumonia (Idiopathic PF, idiopathic non-specific interstitial pneumonia, chronic hypersensitivity pneumonitis, pleuroparenchymal fibroelastosis, unclassifiable interstitial lung disease (ILD)) diagnosed according to the current international guidelines. 3. Age-adjusted peripheral blood leukocyte telomere length < 10th centile on Flow-FISH. 4. FVC > 40% predicted. 5. DLCO > 25% predicted. 6. If receiving background pirfenidone / nintedanib, stable dose for 28 days prior to screening. 7. Able to understand and sign a written informed consent form (or legally authorised representative). 8. Agreement to use a medically approved form of non-hormonal contraception (if of child-bearing potential) (noting that oral contraceptives are advised not to be used concurrently with danazol).

Exclusion Criteria:

1. Actively or imminently listed for lung transplantation. 2. Undergone, awaiting, or likely to require bone marrow transplantation within 12 months. 3. Concurrent enrolment in another study. 4. Females with a positive pregnancy test at screening or currently breastfeeding. 5. Pelvic infection. 6. Past jaundice with oral contraceptives. 7. Undiagnosed abnormal genital bleeding. 8. Undiagnosed ovarian/uterine masses. 9. Any history of malignancy likely to result in significant disability or likely to require significant medical or surgical intervention within the next 12 months. 10. History of androgen-dependent tumour. 11. Any condition other than PF that, in the opinion of the investigator, is likely to result in the death of the participant within the next 12 months. 12. History of end-stage liver disease or ALT or AST > 3 times the upper limit of normal. 13. History of end-stage kidney disease requiring dialysis. 14. Markedly impaired cardiac function. 15. Known increased risk of or history of thromboembolism (e.g. Factor V Leiden, Protein C or S deficiency). 16. Uncontrolled hypertension. 17. Uncontrolled lipoprotein disorder. 18. Poorly-controlled diabetes mellitus. 19. History of marked or persistent androgenic reaction to previous gonadal steroid therapy. 20. History of epilepsy induced or worsened by previous gonadal steroid therapy. 21. History of raised intracranial pressure. 22. Known intolerance to danazol. 23. Porphyria. 24. Use of any of the following agents within 28 days before screening: danazol or other androgen therapy, warfarin or other anticoagulant, carbamazepine, phenytoin, investigational therapy, cytotoxic therapy, tacrolimus, cyclosporine. 25. Professional singer due to potential for voice change. 26. Competitive athletes. 27. Prostate specific antigen (PSA) above the upper limit of normal (adult males only).

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT04638517
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	The University of Queensland
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	Australia
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Pulmonary Fibrosis, Telomere Shortening, Telomere Disease, Dyskeratosis Congenita

Additional Details

TELO-SCOPE is a national, multi-centre, double-blind, placebo-controlled, randomised trial which will be conducted in subjects aged >5 years with a multi-disciplinary diagnosis of pulmonary fibrosis and with age-adjusted telomere length below the 10th centile in adults; and for children (age < 16 years), a confirmed diagnosis of Dyskeratosis Congenita (DC). Consenting participants who meet all other inclusions and no exclusions will be randomised (n=50, 2:1 (danazol:placebo)) to receive danazol (maximum tolerated dose (up to 800mg daily, two-divided doses) or matched placebo, for 12 months in addition to standard of care background therapy. The primary outcome is change in telomere length at 12 months.

Arms & Interventions

Arms

Active Comparator: Danazol

800mg daily in two divided doses orally for 12 months. In subjects who have difficulty tolerating danazol / placebo, the dose will be reduced by 200mg/day and side effects will be reassessed. If symptoms related to the study drug persist, subsequent 200mg/day dose reductions will be allowed until a tolerated dose is achieved. Background antifibrotic therapy is allowed.

Placebo Comparator: Placebo

Matching placebo capsules.

Interventions

Drug: - Danazol

Danazol up to 800mg daily in two-divided doses.

Drug: - Placebo

Matching placebo.

Contact a Trial Team

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