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oai:recercat.cat:11351/89622025-10-24T10:35:23Zcom_2072_378070com_2072_378040col_2072_378092

ePHex: a phase 3, double-blind, placebo-controlled, randomized study to evaluate long-term efficacy and safety of Oxalobacter formigenes in patients with primary hyperoxaluria Collard, Laure Moochhala, Shabbir H. Gould, Edward Boussetta, Abir Fraga Rodriguez, Gloria Mª ABROUG, Saoussen Ariceta Iraola, Gema Institut Català de la Salut [Ariceta G] Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Collard L] Centre Hospitalier Umniversitaire de Liege, Liege, Belgium. [Abroug S] Hôpital Universitaire Sahloul, Sousse, Tunisia. [Moochhala SH] Royal Free Hospital, London, UK. [Gould E] Vanderbilt University Hospital, Nashville, USA. [Boussetta A] Charles Nicolle University Hospital, Tunis, Tunisia. [Fraga G] Vall d’Hebron Hospital Universitari, Barcelona, Spain. Hospital Sant Pau, Barcelona, Spain Vall d'Hebron Barcelona Hospital Campus Malalties congènites - Tractament Bacteris anaerobis Ronyons - Malalties - Tractament ORGANISMS::Bacteria::Gram-Negative Bacteria::Gram-Negative Anaerobic Bacteria::Gram-Negative Anaerobic Straight, Curved, and Helical Rods::Oxalobacter formigenes DISEASES::Congenital, Hereditary, and Neonatal Diseases and Abnormalities::Genetic Diseases, Inborn::Metabolism, Inborn Errors::Carbohydrate Metabolism, Inborn Errors::Hyperoxaluria, Primary Other subheadings::Other subheadings::/therapy ORGANISMOS::Bacteria::bacterias gramnegativas::bacterias anaerobias gramnegativas::bacilos anaerobios gramnegativos, rectos, curvos y espirales::Oxalobacter formigenes ENFERMEDADES::enfermedades y anomalías neonatales congénitas y hereditarias::enfermedades genéticas congénitas::alteraciones congénitas del metabolismo::trastornos congénitos del metabolismo de los carbohidratos::hiperoxaluria primaria Otros calificadores::Otros calificadores::/terapia Oxabact; Oxalobacter formigenes; Primary hyperoxaluria Oxabact; Oxalobacter formigenes; Hiperoxaluria primaria Oxabact; Oxalobacter formigenes; Hiperoxalúria primària Background Primary hyperoxalurias (PHs) are rare genetic diseases that increase the endogenous level of oxalate, a waste metabolite excreted predominantly by the kidneys and also the gut. Treatments aim to improve oxalate excretion, or reduce oxalate generation, to prevent kidney function deterioration. Oxalobacter formigenes is an oxalate metabolizing bacterium. This Phase III, double-blind, placebo-controlled randomized trial investigated the effectiveness of orally administered Oxabact™, a lyophilized O. formigenes formulation, at reducing plasma oxalate levels in patients suffering from PH. Methods Subjects (≥ 2 years of age) with a diagnosis of PH and maintained but suboptimal kidney function (mean estimated glomerular filtration rate at baseline < 90 mL/min/1.73 m2) were eligible to participate. Subjects were randomized to receive Oxabact or placebo twice daily for 52 weeks. Change from baseline in plasma oxalate concentration at Week 52 was the primary study endpoint. Results Forty-three subjects were screened, 25 were recruited and one was discontinued. At Week 52, O. formigenes was established in the gut of subjects receiving Oxabact. Despite decreasing plasma oxalate level in subjects treated with Oxabact, and stable/increased levels with placebo, there was no significant difference between groups in the primary outcome (Least Squares mean estimate of treatment difference was − 3.80 μmol/L; 95% CI: − 7.83, 0.23; p-value = 0.064). Kidney function remained stable in both treatments. Conclusions Oxabact treatment may have stabilized/reduced plasma oxalate versus a rise with placebo, but the difference over 12 months was not statistically significant (p = 0.06). A subtle effect observed with Oxabact suggests that O. formigenes may aid in preventing kidney stones. The study was designed, funded, and managed by OxThera Intellectual Property AB (Stockholm, Sweden). 2023-02-06T09:05:41Z 2023-02-06T09:05:41Z 2023-02 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Ariceta G, Collard L, Abroug S, Moochhala SH, Gould E, Boussetta A, et al. ePHex: a phase 3, double-blind, placebo-controlled, randomized study to evaluate long-term efficacy and safety of Oxalobacter formigenes in patients with primary hyperoxaluria. Pediatr Nephrol. 2023 Feb;38(2):403–15. 1432-198X https://hdl.handle.net/11351/8962 10.1007/s00467-022-05591-5 35552824 000794091100003 http://hdl.handle.net/11351/8962 eng Pediatric Nephrology;38(2) https://doi.org/10.1007/s00467-022-05591-5 Attribution 4.0 International http://creativecommons.org/licenses/by/4.0/ info:eu-repo/semantics/openAccess application/pdf Springer Scientia