Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments

Author:

Litcher-Kelly Leighann,Ozen Ahmet,Ollis Sarah,Feldman Hagit Baris,Yaworsky Andrew,Medrano Paolo,Chongsrisawa Voranush,Brackin Taylor,Perlee Lorah,Walker Marisa,Pradeep Sharanya,Lenardo Michael J.,Harari Olivier A.,Jalbert Jessica J.ORCID

Abstract

Abstract Background CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is ultra-rare (< 100 children or young adults worldwide) and potentially fatal. The study used mixed-methods approaches to assess how pozelimab impacts the signs and symptoms of CHAPLE disease from the patient perspective by combining within-trial interviews and clinical outcome assessments (COAs) (ClinicalTrials.gov, NCT04209634). Methods Interviews conducted with patients/caregivers at screening and week 24 assessed the signs and symptoms of CHAPLE disease, including those which were most bothersome, and evaluated the change. Patients/caregivers and clinicians completed the COAs; interview data contextualized the meaningfulness of change. Results Ten patients (aged 3–19 years) were enrolled; caregivers contributed to nine interviews. Abdominal pain, diarrhea, facial and peripheral edema, nausea, and vomiting are the core signs and symptoms of CHAPLE disease (≥ 90% patients experienced pre-treatment); the most bothersome signs and symptoms were abdominal pain (n = 9) and facial edema (n = 1). All core signs and symptoms were reported as resolved at week 24 interviews. Severity on global assessments changed from “mild” to “very severe” at baseline to “no signs or symptoms” at week 24. Interview results were generally consistent with sign- or symptom-specific COA scores. Conclusions Patients with CHAPLE disease treated with pozelimab for 24 weeks experienced complete resolution of core signs and symptoms. Mixed-methods approaches can contextualize the patient experience (how patients feel and function) in rare disease trials. Trial registration Clinicaltrials.gov, NCT04209634, registered December 24, 2019, https://classic.clinicaltrials.gov/ct2/show/NCT04209634.

Funder

Regeneron Pharmaceuticals

Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health

Publisher

Springer Science and Business Media LLC

Reference23 articles.

1. Ozen A, Comrie WA, Ardy RC, Domínguez Conde C, Dalgic B, Beser Ö, et al. CD55 deficiency, early-onset protein-losing enteropathy, and thrombosis. N Engl J Med. 2017;377(1):52–61.

2. Kurolap A, Eshach Adiv O, Hershkovitz T, Tabib A, Karbian N, Paperna T, et al. Eculizumab is safe and effective as a long-term treatment for protein-losing enteropathy due to CD55 deficiency. J Pediatr Gastroenterol Nutr. 2019;68(3):325–33.

3. Comrie WA, Ozen A, Ardy RC, Su H, Boztug K, Lenardo M. Inherited CD55 Deficiency in patients with early onset protein-losing enteropathy and thrombosis. J Immunol. 2017;198(1Supplement):5910–5910.

4. Regeneron Pharmaceuticals Inc. Pozelimab (C5 antibody) BLA for treatment of children and adults with ultra-rare CHAPLE disease accepted for FDA priority review. 2023. https://investor.regeneron.com/news-releases/news-release-details/pozelimab-c5-antibody-bla-treatment-children-and-adults-ultra. Accessed 12 Oct 2023.

5. ClinicalTrials.gov. Open-label efficacy and safety study of pozelimab in patients with CD55-deficient protein-losing enteropathy (CHAPLE disease). 2019. https://clinicaltrials.gov/ct2/show/NCT04209634. Accessed 17 May 2023.

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