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TELO-SCOPE study: a randomised, double-blind, placebo-controlled, phase 2 trial of danazol for short telomere related pulmonary fibrosis

Citation

Mackintosh, JA and Pietsch, M and Lutzky, V and Enever, D and Bancroft, S and Apte, SH and Tan, M and Yerkovich, ST and Dickinson, JL and Pickett, HA and Selvadurai, H and Grainge, C and Goh, NS and Hopkins, P and Glaspole, I and Reynolds, PN and Wrobel, J and Jaffe, A and Corte, TJ and Chambers, DC, TELO-SCOPE study: a randomised, double-blind, placebo-controlled, phase 2 trial of danazol for short telomere related pulmonary fibrosis, BMJ Open Respiratory Research, 8, (1) pp. 1-8. ISSN 2052-4439 (2021) [Refereed Article]


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Author(s) (or their employer(s)) 2021. Re-use permitted under the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.commercial re-use. See rights and permissions. Published by BMJ

DOI: doi:10.1136/bmjresp-2021-001127

Abstract

Introduction: Recent discoveries have identified shortened telomeres and related mutations in people with pulmonary fibrosis (PF). There is evidence to suggest that androgens, including danazol, may be effective in lengthening telomeres in peripheral blood cells. This study aims to assess the safety and efficacy of danazol in adults and children with PF associated with telomere shortening.

Methods and analysis: A multi-centre, double-blind, placebo-controlled, randomised trial of danazol will be conducted in subjects aged >5 years with PF associated with age-adjusted telomere length ≤10th centile measured by flow fluorescence in situ hybridisation; or in children, a diagnosis of dyskeratosis congenita. Adult participants will receive danazol 800 mg daily in two divided doses or identical placebo capsules orally for 12 months, in addition to standard of care (including pirfenidone or nintedanib). Paediatric participants will receive danazol 2 mg/kg/day orally in two divided doses or identical placebo for 6 months. If no side effects are encountered, the dose will be escalated to 4 mg/kg/day (maximum 800 mg daily) orally in two divided doses for a further 6 months. The primary outcome is change in absolute telomere length in base pairs, measured using the telomere shortest length assay (TeSLA), at 12 months in the intention to treat population.

Item Details

Item Type:Refereed Article
Keywords:pulmonary fibrosis, familial, inherited predisposition
Research Division:Biomedical and Clinical Sciences
Research Group:Cardiovascular medicine and haematology
Research Field:Respiratory diseases
Objective Division:Health
Objective Group:Clinical health
Objective Field:Treatment of human diseases and conditions
UTAS Author:Dickinson, JL (Professor Joanne Dickinson)
ID Code:148200
Year Published:2021
Deposited By:Menzies Institute for Medical Research
Deposited On:2021-12-10
Last Modified:2022-01-14
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