The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform

Janet Sultana; Nikita Camilleri; Salvatore Crisafulli; John Joseph Borg; Silvan Spagnol; Silvia Tillati; Joseph Borg

doi:10.1177/26330040231213888

The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform

Ther Adv Rare Dis. 2023 Dec 18:4:26330040231213888. doi: 10.1177/26330040231213888. eCollection 2023 Jan-Dec.

Authors

Janet Sultana^{1

2

3}, Nikita Camilleri³, Salvatore Crisafulli⁴, John Joseph Borg⁵, Silvan Spagnol², Silvia Tillati⁶, Joseph Borg³

Affiliations

¹ Exeter College of Medicine and Health, St. Luke's Campus, Exeter, EX4 4QJ, UK.
² Quality Improvement, Pharmacy Department, Mater Dei Hospital, Msida, Malta.
³ Applied Biomedical Science, Faculty of Health Sciences, Mater Dei Medical School, Msida, Malta.
⁴ Department of Medicine, University of Verona, Verona, Italy.
⁵ Post-licensing Department, Malta Medicines Authority, Msida, Malta.
⁶ Unit of Medical Statistics, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.

Abstract

To date, there is no published overview of the drug pipeline in granulomatosis with polyangiitis (GPA), a rare disease. The aim of this study was to identify clinical trials from two study repositories. A review of clinical trials was conducted using publicly available data. Clinicaltrials.gov and International Clinical Trials Registry Platform were searched from inception until 25 September 2022. Only GPA-specific studies were included; these were described in detail. A total of 137 studies were identified in the trial repositories, of which 108 (79%) studies were found to concern GPA. Of these 108 studies, 67 enrolled GPA patients to investigate pharmacotherapy in this disease (62%). Most studies included all severity types (n = 51; 76%); the scope of almost half of the studies was remission induction (n = 33; 49%). The drug class which was by the most widely investigated in trials was the non-corticosteroid immunosuppressant drug class (46; 68.7%), monoclonal antibodies (32; 47.8%), and corticosteroids (31; 46.3%). There is a need for more GPA trials to generate evidence on effectiveness in terms of severity-specificity and maintenance of remission.

Keywords: clinical trials; granulomatosis with polyangiitis; pharmacotherapy.

Plain language summary

The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials To date, there is no published overview of the drug pipeline in granulomatosis with polyangiitis (referred to in this paper as GPA), a rare disease. The aim of this study was to identify such studies from two study archives. Clinicaltrials.gov and International Clinical Trials Registry Platform (ICTRP) were searched from inception until 25th September 2022. Studies recruiting GPA patients were included; these were described in detail. A total of 137 studies were identified in the trial repositories, of which 108 were found to concern GPA. Of these 108 studies, 67 enrolled GPA patients to investigate the treatment of this disease through the administration of drugs. Most studies included all severity types (n = 51); the scope of almost half of the studies was to induce remission (n = 33). The drug classes which were the most widely investigated in trials were non-corticosteroid immunosuppressant drugs (n = 46), monoclonal antibodies (n = 32), and corticosteroids (n = 31). There is a need for more GPA clinical trials to generate evidence on effectiveness of drugs in terms of severity-specificity and maintenance of remission.

Publication types

Review