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https://hdl.handle.net/2440/134267
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Type: | Journal article |
Title: | TELO-SCOPE study: a randomised, double-blind, placebo-controlled, phase 2 trial of danazol for short telomere related pulmonary fibrosis |
Author: | MacKintosh, J.A. Pietsch, M. Lutzky, V. Enever, D. Bancroft, S. Apte, S.H. Tan, M. Yerkovich, S.T. Dickinson, J.L. Pickett, H.A. Selvadurai, H. Grainge, C. Goh, N.S. Hopkins, P. Glaspole, I. Reynolds, P.N. Wrobel, J. Jaffe, A. Corte, T.J. Chambers, D.C. |
Citation: | BMJ Open Respiratory Research, 2021; 8(1):e001127-1-e001127-8 |
Publisher: | BMJ Publishing Group |
Issue Date: | 2021 |
ISSN: | 2052-4439 2052-4439 |
Statement of Responsibility: | John A Mackintosh, Maria Pietsch, Viviana Lutzky, Debra Enever, Sandra Bancroft, Simon H Apte, Maxine Tan, Stephanie T Yerkovich, Joanne L Dickinson, Hilda A Pickett, Hiran Selvadurai, Christopher Grainge, Nicole S Goh, Peter Hopkins, Ian Glaspole, Paul N Reynolds, Jeremy Wrobel, Adam Jaffe, Tamera J Corte, Daniel C Chambers |
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. Ethics and dissemination: Ethics approval has been granted in Australia by the Metro South Human Research Ethics Committee (HREC/2020/QMS/66385). The study will be conducted and reported according to Standard Protocol Items: Recommendations for Interventional Trials guidelines. Results will be published in peer-reviewed journals and presented at international and national conferences. |
Keywords: | Pulmonary fibrosis |
Rights: | © Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. http://creativecommons.org/licenses/by-nc/4.0/. This is an open access article distributed in accordance with 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/. |
DOI: | 10.1136/bmjresp-2021-001127 |
Grant ID: | http://purl.org/au-research/grants/nhmrc/1199373 |
Published version: | http://dx.doi.org/10.1136/bmjresp-2021-001127 |
Appears in Collections: | Medicine publications |
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hdl_134267.pdf | Published version | 321.14 kB | Adobe PDF | View/Open |
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