Spironolactone for Adult Female Acne (SAFA): Protocol for a double-blind, placebo-controlled, phase III randomised study of spironolactone as systemic therapy for acne in adult women

Susanne Renz, Fay Chinnery, Beth Stuart, Laura Day , Ingrid Muller , Irene Soulsby , Jacqueline Nuttall , Karen Thomas, Kim S Thomas , Tracey Sach , Louise Stanton, Matthew J Ridd, Nick Francis, Paul Little , Miriam Santer*

*Corresponding author for this work

Research output: Contribution to journalArticle (Academic Journal)peer-review

Abstract

Introduction Acne is one of the most common inflammatory skin diseases worldwide and can have significant psychosocial impact and cause permanent scarring. Spironolactone, a potassium-sparing diuretic, has antiandrogenic properties, potentially reducing sebum production and hyperkeratinisation in acne-prone follicles. Dermatologists have prescribed spironolactone for acne in women for over 30 years, but robust clinical study data are lacking. This study seeks to evaluate whether spironolactone is clinically effective and cost-effective in treating acne in women.

Methods and analysis Women (≥18 years) with persistent facial acne requiring systemic therapy are randomised to receive one tablet per day of 50 mg spironolactone or a matched placebo until week 6, increasing to up to two tablets per day (total of 100 mg spironolactone or matched placebo) until week 24, along with usual topical therapy if desired. Study treatment stops at week 24; participants are informed of their treatment allocation and enter an unblinded observational follow-up period for up to 6 months (up to week 52 after baseline). Primary outcome is the Acne-specific Quality of Life (Acne-QoL) symptom subscale score at week 12. Secondary outcomes include Acne-QoL total and subscales; participant acne self-assessment recorded on a 6-point Likert scale at 6, 12, 24 weeks and up to 52 weeks; Investigator’s Global Assessment at weeks 6 and 12; cost and cost effectiveness are assessed over 24 weeks. Aiming to detect a group difference of 2 points on the Acne-QoL symptom subscale (SD 5.8, effect size 0.35), allowing for 20% loss to follow-up, gives a sample size of 398 participants.

Ethics and dissemination This protocol was approved by Wales Research Ethics Committee (18/WA/0420). Follow-up to be completed in early 2022. Findings will be disseminated to participants, peer-reviewed journals, networks and patient groups, on social media, on the study website and the Southampton Clinical Trials Unit website to maximise impact.

Trial registration number ISRCTN12892056;Pre-results.
Original languageEnglish
Article numbere053876
Number of pages13
JournalBMJ Open
Volume11
Issue number8
DOIs
Publication statusPublished - 26 Aug 2021

Bibliographical note

Funding Information:
Funding This project is funded by the National Institute for Health Research (NIHR) Health Technology Assessment (HTA) programme (grant reference number 16/13/02) and supported by NIHR CTU support funding at Southampton CTU. The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care. The NIHR HTA funder will play no role in the execution, analysis, interpretation of data or study publication. The study is registered on the UK NIHR study portfolio, meaning there are research nurses based at UK hospitals who help in screening potential patients to identify those eligible for the study. Southampton CTU, an NIHR CTU support funded UK Clinical Research Collaboration registered CTU, is coordinating the study. University of Southampton is the sponsor for the study. Competing interests None declared. Patient consent for publication Not required.

Funding Information:
Twitter Ingrid Muller @IngridMuller7, Karen Thomas @kazthomas, Matthew J Ridd @riddmj and Nick Francis @nickafrancis Acknowledgements The authors thank the virtual patient panel (brought together by NIHR INVOLVE) for their advice on the study design. The study was developed with support from the UK Dermatology Clinical Trials Network (UK DCTN). The UK DCTN is grateful to the British Association of Dermatologists and the University of Nottingham for financial support of the Network. UK DCTN conducted surveys among patients with acne and health professionals managing acne to inform the study design, promoting the study and identifying and advising on potential hospital sites delivering the study. The authors also thank Wessex CRN for funding the initial social media advertising campaign; Jessica Boxall and Liz Allaway for management of the study social media accounts as well as running and coordinating the social media adverts; hospital dermatology centres recruiting for the study: Queen Elizabeth Hospital, Birmingham; Bristol Royal Infirmary Dermatology Centre, Bristol; University Hospital of Wales, Cardiff; General Hospital, Epsom; District Hospital, Harrogate; St Mary’s Hospital (Imperial College NHS Healthcare Trust), London; Queen’s Medical Centre, Nottingham; General Hospital, Poole; St Mary’s General Hospital Dermatology Centre, Portsmouth; Swansea Bay University Health Board, Swansea; participant identification centres (PICs) for searching their patient lists and mail outs and clinical research networks for helping to identify potential PICs.

Publisher Copyright:
© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY. Published by BMJ.

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