A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol

Abstract Background Graves’ orbitopathy (GO) is a prevalent manifestation of Graves’ disease (GD), characterized by proptosis, eyelid retraction, soft tissue swelling, diplopia, and potential visual acuity impairment. Furthermore, even mild GO can significantly impact mental health and overall quali...

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Main Authors: Chia-Hung Lin, Yi-Hsuan Wei, Jin-Ying Lu, Hung-Yuan Li, Chung-Wei Lee, Chung-Yi Yang, Chin-Hao Chang, Wan-Chen Wu, Chih-Yuan Wang, Shyang-Rong Shih
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Language:English
Published: BMC 2025-08-01
Series:Trials
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Online Access:https://doi.org/10.1186/s13063-025-09002-6
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author Chia-Hung Lin
Yi-Hsuan Wei
Jin-Ying Lu
Hung-Yuan Li
Chung-Wei Lee
Chung-Yi Yang
Chin-Hao Chang
Wan-Chen Wu
Chih-Yuan Wang
Shyang-Rong Shih
author_facet Chia-Hung Lin
Yi-Hsuan Wei
Jin-Ying Lu
Hung-Yuan Li
Chung-Wei Lee
Chung-Yi Yang
Chin-Hao Chang
Wan-Chen Wu
Chih-Yuan Wang
Shyang-Rong Shih
author_sort Chia-Hung Lin
collection DOAJ
description Abstract Background Graves’ orbitopathy (GO) is a prevalent manifestation of Graves’ disease (GD), characterized by proptosis, eyelid retraction, soft tissue swelling, diplopia, and potential visual acuity impairment. Furthermore, even mild GO can significantly impact mental health and overall quality of life for patients with GD. Despite its severity, available medical treatments for mild GO are limited. Recent basic studies on orbital fibroblasts suggest hydroxychloroquine (HCQ) as a promising therapeutic agent for GO patients. This randomized controlled trial (RCT) was designed to assess the efficacy of HCQ in treating mild GO.  Methods This multi-center open-label RCT will be conducted in Taiwan with a total of 108 participants randomized into HCQ and control groups at 2:1 allocation ratio. The primary endpoint of this study is a composite outcome of ophthalmic parameters including eyelid aperture, soft tissue involvement, and exophthalmos. Secondary endpoints comprise changes in quality of life (GO-QoL), orbital volumetry via computed tomography (CT), diplopia scores, clinical activity scores (CAS), visual acuity, and thyroid autoantibodies. Discussion This RCT will elucidate the clinical benefits of oral HCQ in patients with mild GO, assessing ophthalmic outcomes, quality of life, disease activity, and thyroid autoantibodies. In addition, data obtained from orbital CT measurements will provide valuable insights into subtle changes in orbital fat and extra-ocular muscle volumes, potentially offering an objective tool for monitoring GO progression. Trial registration {2a, 2b} ClinicalTrials.gov NCT05126147. Registered on November 2021.  https://clinicaltrials.gov/study/NCT05126147 . All items from the World Health Organization (WHO) Trial Registration Data Set are addressed within the relevant sections of this protocol.
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spelling doaj-art-dc13d951f9f644b9a975adf367f21e132025-08-20T03:46:20ZengBMCTrials1745-62152025-08-012611910.1186/s13063-025-09002-6A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocolChia-Hung Lin0Yi-Hsuan Wei1Jin-Ying Lu2Hung-Yuan Li3Chung-Wei Lee4Chung-Yi Yang5Chin-Hao Chang6Wan-Chen Wu7Chih-Yuan Wang8Shyang-Rong Shih9Division of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University HospitalDepartment of Ophthalmology, National Taiwan University HospitalDivision of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University HospitalDivision of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University HospitalDepartment of Medical Imaging, National Taiwan University HospitalDepartment of Medical Imaging, I-Shou UniversityDepartment of Medical Research, National Taiwan University HospitalDivision of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University HospitalDivision of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University HospitalDivision of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University HospitalAbstract Background Graves’ orbitopathy (GO) is a prevalent manifestation of Graves’ disease (GD), characterized by proptosis, eyelid retraction, soft tissue swelling, diplopia, and potential visual acuity impairment. Furthermore, even mild GO can significantly impact mental health and overall quality of life for patients with GD. Despite its severity, available medical treatments for mild GO are limited. Recent basic studies on orbital fibroblasts suggest hydroxychloroquine (HCQ) as a promising therapeutic agent for GO patients. This randomized controlled trial (RCT) was designed to assess the efficacy of HCQ in treating mild GO.  Methods This multi-center open-label RCT will be conducted in Taiwan with a total of 108 participants randomized into HCQ and control groups at 2:1 allocation ratio. The primary endpoint of this study is a composite outcome of ophthalmic parameters including eyelid aperture, soft tissue involvement, and exophthalmos. Secondary endpoints comprise changes in quality of life (GO-QoL), orbital volumetry via computed tomography (CT), diplopia scores, clinical activity scores (CAS), visual acuity, and thyroid autoantibodies. Discussion This RCT will elucidate the clinical benefits of oral HCQ in patients with mild GO, assessing ophthalmic outcomes, quality of life, disease activity, and thyroid autoantibodies. In addition, data obtained from orbital CT measurements will provide valuable insights into subtle changes in orbital fat and extra-ocular muscle volumes, potentially offering an objective tool for monitoring GO progression. Trial registration {2a, 2b} ClinicalTrials.gov NCT05126147. Registered on November 2021.  https://clinicaltrials.gov/study/NCT05126147 . All items from the World Health Organization (WHO) Trial Registration Data Set are addressed within the relevant sections of this protocol.https://doi.org/10.1186/s13063-025-09002-6Graves’ orbitopathyThyroid eye diseaseGraves’ diseaseHydroxychloroquine
spellingShingle Chia-Hung Lin
Yi-Hsuan Wei
Jin-Ying Lu
Hung-Yuan Li
Chung-Wei Lee
Chung-Yi Yang
Chin-Hao Chang
Wan-Chen Wu
Chih-Yuan Wang
Shyang-Rong Shih
A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol
Trials
Graves’ orbitopathy
Thyroid eye disease
Graves’ disease
Hydroxychloroquine
title A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol
title_full A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol
title_fullStr A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol
title_full_unstemmed A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol
title_short A randomized controlled trial on the effect of hydroxychloroquine in mild Graves’ orbitopathy (GO-HCQ): study protocol
title_sort randomized controlled trial on the effect of hydroxychloroquine in mild graves orbitopathy go hcq study protocol
topic Graves’ orbitopathy
Thyroid eye disease
Graves’ disease
Hydroxychloroquine
url https://doi.org/10.1186/s13063-025-09002-6
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