PATIENT-REPORTED OUTCOMES IN THE OPEN-LABEL EXTENSION OF THE CHAPTER-1 STUDY ASSESSING LONG-TERM PROPHYLACTIC TREATMENT WITH ORAL DEUCRICTIBANT IN PARTICIPANTS WITH HEREDITARY ANGIOEDEMA
Author(s)
Markus Magerl, MD1, John Anderson, MD2, Francesco Arcoleo, MD3, Mauro Cancian, MD, PhD4, Hugo Chapdelaine, MD, FRCPC5, Niall Conlon, PhD, FRCPath6, Efrem Eren, MBBS, MRCP, FRCPath, PhD7, Mark Gompels, MD8, Sofia Grigoriadou, MD9, Maria D. Guarino, MD, PhD10, Padmalal Gurugama, MBBS, MSc, MD, MRCP, FRCPath11, Sorena Kiani, BSc MBBS PhD FRCP FRCPath12, Tamar Kinaciyan, MD13, Michael E. Manning, MD14, Marcin Stobiecki, PhD, MD15, Michael D. Tarzi, MA, MD, MRCP, FRCPath16, Anna Valerieva, MD, PhD17, H. James Wedner, MD, FACP, FAAAAI18, William H. Yang, MD, FRCPC, FAAAAI19, Andrea Zanichelli, PhD, MD20, Rafael Crabbé, MD21, Susan Mulders, PhD22, Jonathan Levy, PhD23, Ulrich Freudensprung, MS24, Umar Katbeh, MA, PhD24, Jochen Knolle, PhD25, Anne Lesage, PhD26, Peng Lu, MD, PhD23, Emel Aygören-Pürsün, MD27, Marc A. Riedl, MD28.
1Institute of Allergology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin and Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany, 2AllerVie Health, Clinical Research Center of Alabama, Birmingham, AL, USA, 3AOR Villa Sofia-Cervello, UOC di Patologia Clinica e Immunologia, Palermo, Italy, 4Azienda Ospedale Università di Padova, Padua, Italy, 5CHU de Montréal, Université de Montréal, Montréal, QC, Canada, 6St. James's Hospital and Trinity College, Wellcome Trust CRF, Dublin, Ireland, 7University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom, 8North Bristol NHS Trust, Bristol, United Kingdom, 9Barts Health NHS Trust, Department of Immunology, London, United Kingdom, 10U.O.C. Allergologia Ospedale di Civitanova Marche, Civitanova Marche, Italy, 11Cambridge University Hospitals NHS Foundation Trust, Department of Clinical Immunology, Cambridge, United Kingdom, 12Royal Free London NHS Foundation Trust, Department of Immunology, London, United Kingdom, 13Medical University of Vienna, Department of Dermatology, Vienna, Austria, 14Allergy, Asthma and Immunology Associates, Ltd., Scottsdale, AZ, USA, 15Jagiellonian University Medical College, Department of Clinical and Environmental Allergology, Kraków, Poland, 16University Hospitals Sussex NHS Foundation Trust, Department of Respiratory Medicine, Brighton, United Kingdom, 17Medical University of Sofia, Department of Allergology, Sofia, Bulgaria, 18Washington University School of Medicine, Division of Allergy and Immunology, Department of Medicine, St. Louis, MO, USA, 19University of Ottawa, Ottawa Allergy Research Corporation, Department of Medicine, Ottawa, ON, Canada, 20Universita degli Studi di Milano, Dipartimento di Scienze Biomediche per la Salute; IRCCS Policlinico San Donato, UO Medicina, Centro Angioedema, San Donato Milanese, Milano, Italy, 21RC Consultancy, Bassins, Switzerland, 22Mulders Clinical Consulting, Groesbeek, Netherlands, 23Pharvaris Inc., Lexington, MA, USA, 24Pharvaris GmbH, Zug, Switzerland, 25JCK Consult, Frankfurt am Main, Germany, 26GrayMatters Consulting, Schilde, Belgium, 27Goethe University Frankfurt, Department of Pediatrics, Frankfurt am Main, Germany, 28University of California San Diego, Division of Allergy and Immunology, La Jolla, CA, USA.
1Institute of Allergology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin and Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany, 2AllerVie Health, Clinical Research Center of Alabama, Birmingham, AL, USA, 3AOR Villa Sofia-Cervello, UOC di Patologia Clinica e Immunologia, Palermo, Italy, 4Azienda Ospedale Università di Padova, Padua, Italy, 5CHU de Montréal, Université de Montréal, Montréal, QC, Canada, 6St. James's Hospital and Trinity College, Wellcome Trust CRF, Dublin, Ireland, 7University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom, 8North Bristol NHS Trust, Bristol, United Kingdom, 9Barts Health NHS Trust, Department of Immunology, London, United Kingdom, 10U.O.C. Allergologia Ospedale di Civitanova Marche, Civitanova Marche, Italy, 11Cambridge University Hospitals NHS Foundation Trust, Department of Clinical Immunology, Cambridge, United Kingdom, 12Royal Free London NHS Foundation Trust, Department of Immunology, London, United Kingdom, 13Medical University of Vienna, Department of Dermatology, Vienna, Austria, 14Allergy, Asthma and Immunology Associates, Ltd., Scottsdale, AZ, USA, 15Jagiellonian University Medical College, Department of Clinical and Environmental Allergology, Kraków, Poland, 16University Hospitals Sussex NHS Foundation Trust, Department of Respiratory Medicine, Brighton, United Kingdom, 17Medical University of Sofia, Department of Allergology, Sofia, Bulgaria, 18Washington University School of Medicine, Division of Allergy and Immunology, Department of Medicine, St. Louis, MO, USA, 19University of Ottawa, Ottawa Allergy Research Corporation, Department of Medicine, Ottawa, ON, Canada, 20Universita degli Studi di Milano, Dipartimento di Scienze Biomediche per la Salute; IRCCS Policlinico San Donato, UO Medicina, Centro Angioedema, San Donato Milanese, Milano, Italy, 21RC Consultancy, Bassins, Switzerland, 22Mulders Clinical Consulting, Groesbeek, Netherlands, 23Pharvaris Inc., Lexington, MA, USA, 24Pharvaris GmbH, Zug, Switzerland, 25JCK Consult, Frankfurt am Main, Germany, 26GrayMatters Consulting, Schilde, Belgium, 27Goethe University Frankfurt, Department of Pediatrics, Frankfurt am Main, Germany, 28University of California San Diego, Division of Allergy and Immunology, La Jolla, CA, USA.
OBJECTIVES: Hereditary angioedema (HAE), a rare bradykinin-mediated disease, is characterized by recurrent, unpredictable, debilitating, and painful swelling attacks, which negatively impact patients’ health-related quality of life (HRQoL). Deucrictibant is a potent, selective, orally administered bradykinin B2 receptor antagonist under development for both prophylaxis and on-demand treatment of attacks of bradykinin-mediated angioedema, including HAE. Long-term safety and efficacy of deucrictibant for prophylaxis of HAE attacks were evaluated in the open-label extension (OLE) of the two-part Phase 2 CHAPTER-1 (NCT05047185) study. Final results on patient-reported disease control, HRQoL measures, and treatment satisfaction from the OLE are presented here.
METHODS: Adults aged ≥18 and ≤75 years with HAE-1/2 were enrolled in CHAPTER-1. Of the 34 enrolled participants, 30 completed the 12-week randomized controlled trial (RCT) and continued into the OLE. Participants self-administered deucrictibant 40 mg/day during the OLE. Disease control was assessed using the 4-week Angioedema Control Test (AECT-4wk); HRQoL using the Angioedema QoL Questionnaire (AE-QoL) and Patient Global Assessment of Change (PGA-Change); and treatment satisfaction using the 11-item Treatment Satisfaction Questionnaire for Medication (TSQM-II).
RESULTS: During the OLE (weeks 18-134), at least 96% of participants achieved well-controlled HAE (AECT score ≥10) and mean AECT score remained >14. Improvements in HRQoL observed in the RCT were sustained across weeks 18-134: mean AE-QoL total scores ranged between 14.2-23.5 and >80% of participants reported feeling “much better” on the PGA-Change compared with RCT baseline. Across weeks 18-134, mean TSQM-II global satisfaction scores ranged between 80.9-90.7; domain scores for “effectiveness” (82.1-92.2), “side effects” (96.7-100), and “convenience” (72.0-78.9) were also consistently high in the OLE.
CONCLUSIONS: Final results from the CHAPTER-1 OLE study provide further evidence on the effects of long-term treatment with oral deucrictibant for prophylaxis of HAE attacks on disease control, HRQoL, and treatment satisfaction in participants with HAE.
METHODS: Adults aged ≥18 and ≤75 years with HAE-1/2 were enrolled in CHAPTER-1. Of the 34 enrolled participants, 30 completed the 12-week randomized controlled trial (RCT) and continued into the OLE. Participants self-administered deucrictibant 40 mg/day during the OLE. Disease control was assessed using the 4-week Angioedema Control Test (AECT-4wk); HRQoL using the Angioedema QoL Questionnaire (AE-QoL) and Patient Global Assessment of Change (PGA-Change); and treatment satisfaction using the 11-item Treatment Satisfaction Questionnaire for Medication (TSQM-II).
RESULTS: During the OLE (weeks 18-134), at least 96% of participants achieved well-controlled HAE (AECT score ≥10) and mean AECT score remained >14. Improvements in HRQoL observed in the RCT were sustained across weeks 18-134: mean AE-QoL total scores ranged between 14.2-23.5 and >80% of participants reported feeling “much better” on the PGA-Change compared with RCT baseline. Across weeks 18-134, mean TSQM-II global satisfaction scores ranged between 80.9-90.7; domain scores for “effectiveness” (82.1-92.2), “side effects” (96.7-100), and “convenience” (72.0-78.9) were also consistently high in the OLE.
CONCLUSIONS: Final results from the CHAPTER-1 OLE study provide further evidence on the effects of long-term treatment with oral deucrictibant for prophylaxis of HAE attacks on disease control, HRQoL, and treatment satisfaction in participants with HAE.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO175
Topic
Clinical Outcomes
Topic Subcategory
Relating Intermediate to Long-term Outcomes
Disease
Rare & Orphan Diseases