ABS0496 GLOBAL PATTERNS AND CHALLENGES IN PATIENT ENROLMENT IN IDIOPATHIC INFLAMMATORY MYOPATHY (IIM) CLINICAL TRIALS – RESULTS FROM THE 2024 MCTC INVESTIGATORS' SURVEY

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Background: The Myositis Clinical Trials Consortium (MCTC) is an international collaboration of clinical investigators, research professionals, medical centres, patient groups, and industry stakeholders allied in advancing clinical trials in idiopathic inflammatory myopathies (IIM). Despite a recent increase in IIM trials, most are performed at only few select centres, creating a current landscape which limits subjects' diversity and access to trials. Objectives: To identify global patterns in clinical trial enrolment among IIM physicians. Methods: The online 2024 Myositis Investigators' Survey was broadly distributed worldwide to IIM-focused physicians joining the MCTC via professional society and peer-to-peer e-mail invitations and through existing MCTC membership rolls. Data was securely collected/stored, and respondents consented to use of anonymized data for research analyses. The survey collected data on the investigators' background, clinical practice, and research experience. Information both about IIM patients seen for clinical care in the previous year and those recruited into clinical trials (including pilot, open-label, and phase 2/3/4) over the previous three years was recorded. Survey responses were collected from July to December 2024. Fisher's exact test was used to compare different regions. Results: A total of 382 investigators from 42 countries/regions participated in the survey. Patient volume and recruitment data was analysed in 301 investigators after excluding 81 with missing information on patient load and recruitment. Among these 301 respondents, 92 (30.6%) were from Asia-Pacific, 78 (25.9%) from Europe, 71 (23.6%) from North America, 50 (16.6%) from Central/ South America, 7 (2.3%) from the Middle East, and 3 (1.0%) from Africa. The majority (81.7%) treated adult IIM patients, 8.3% treated children, and 7.0% treated both. Most investigators specialized in rheumatology (80%), with the remaining in neurology (7%), pulmonology (7%) and dermatology (4.7%). Investigators had a median of 10 years (IQR: 5 – 18) of clinical research experience, with 65.8% reporting previous experience in IIM clinical research. Dermatomyositis (DM) and anti-synthetase syndrome (ASSD) were the most commonly reported IIM subtypes (Figure 1), with 89% and 86% of investigators, respectively, seeing at least one DM or ASSD patient in the previous year. 113 investigators (37.5%) had recruited patients into at least one IIM clinical trial in the previous three years. Almost 30% of investigators had recruited at least one DM patient into clinical trials, but only 11.6% had recruited more than three patients over that period. Amongst "high-volume" sites who saw more than 20 DM patients in the past year, only 35.1% had enrolled more than three DM patients into a clinical trial in the last 3 years (and 42.6% had not enrolled any). Enrolment rates were lower in other IIM subtypes (ASSD: 17.6%, immune-mediated necrotising myopathy: 11.6%, polymyositis: 10.0%, clinically amyopathic DM: 6.3%, inclusion body myositis: 6%) (Figure 1&2). Among IIM physicians who treated paediatric patients, 4 of 46 (8.7%) had enrolled at least one patient into clinical trials in the previous three years. IIM-associated with interstitial lung disease (IIM-ILD) was encountered by 90.7% of investigators, with 45.2% of investigators seeing more than 10 cases in the previous year. However, only 15.9% had enrolled IIM-ILD patients into clinical trials over the past three years. The proportion of investigators recruiting patients into clinical trials was higher in Europe (41/78, 52.6%), North America (33/71, 46.5%), and the Middle East (3/7, 42.9%) compared to Central/South America (13/50, 26.0%), Asia-Pacific (23/92, 25.0%) and Africa (0/3, 0%) (p<0.001). Recruitment rates were qualitatively similar between rheumatologists and other subspecialists. Conclusion: The 2024 MCTC Investigators' survey reveals significant regional disparities in IIM patient recruitment for clinical trials, with lower rates from regions outside Europe and North America. Moreover, despite extensive investigator clinical research experience and IIM patient volumes, enrolment remains suboptimal, particularly for non-DM IIM subtypes and paediatric patients. These findings underscore the need for targeted strategies to enhance global trial participation and patients' access to IIM clinical trials. REFERENCES: NIL . Acknowledgements: 1. The authors wish to thank the MCTC steering committee members and all MCTC investigators for their contribution and participation in the Myositis Investigator Member Information Survey. 2. NIAMS IRP (HK). Disclosure of Interests: Iris Yan Ki Tang: None declared, Shiri Keret: None declared, Hanna Kim Expert panel for Cabaletta Panel, previous CRADA with Eli Lilly and Company providing study support and drug (baricitinib), Bristol Myers Squibb – providing drug for research study, Akira Yoshida: None declared, Eduardo Dourado: None declared, Edoardo Conticini Speaking honoraria from GSK, Eli Lilly, Chiesi and Dr Schar, Océane Landon-Cardinal: None declared, Kaveh Ardalan Dr. Ardalan has received consulting fees and served on a scientific advisory board for Cabaletta Bio, and is Vice Chair of the Childhood Arthritis and Rheumatology Research Alliance Juvenile Dermatomyositis Committee (paid consultant position), Anuradha Bishnoi: None declared, Faye Pais: None declared, Sabeena Y Usman: None declared, Solciris Dominguez: None declared, Gayathri Tulluru: None declared, Chengappa Kavadichanda: None declared, Daphne Rivero Gallegos: None declared, Francisca Bozan: None declared, James B. Lilleker: None declared, Kaushik Sreerama Reddy: None declared, Rachid Smaili: None declared, Thomas Khoo: None declared, Ting-Yuan Lan: None declared, Valérie Leclair: None declared, Marc Jansen: None declared. Belina Yi: None declared, Silvia Rosina: None declared, Chester V Oddis: None declared. Jiří Vencovský: None declared, Masataka Kuwana Speaking honoraria from Boehringer-Ingelheim, royalties from MBL, Consulting Honoraria from Argenx, Boehringer-Ingelheim, and GSK, Research grants from Boehringer-Ingelheim & MBL, Lisa Rider Hope Pharmaceuticals: grant/contract, Prateek C. Gandiga Honoraria for consultancy from Kezar Pharmaceuticals and Janssen Pharmaceuticals, Rohit Aggarwal Dr. Rohit Aggarwal has received consulting fees from Alexion, ANI Pharmaceutical, Argenx, Artasome, AstraZeneca, Boehringer Ingelheim, Bristol Myers-Squibb, CabalettaBio, Capella, Capstan, Corbus, CSL Behring, EMD Serono, Galapagos, Horizontal Therapeutics, I-Cell, Immunovant, Janssen, Kezar, Kyverna, Lilly, Manta Medicines Corporation, Merck, Novartis, Nuvig Therapeutic, Octapharma, Pfizer, Roivant, Sanofi, Teva, Tourmaline Bio, and Verismo Therapeutics. Dr. Rohit Aggarwal has received research grants from Boehringer Ingelheim, Bristol-Myers Squibb, EMD Serono, Janssen, Mallinckrodt, Pfizer, and Q32. © The Authors 2025. This abstract is an open access article published in Annals of Rheumatic Diseases under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). Neither EULAR nor the publisher make any representation as to the accuracy of the content. The authors are solely responsible for the content in their abstract including accuracy of the facts, statements, results, conclusion, citing resources etc.

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