In a phase 3, placebo-controlled trial of adults with primary immunoglobulin A (IgA) nephropathy, a formulation of targeted-release budesonide (Nefecon) reduced the decline in estimated glomerular filtration rates (eGFRs) and reduced proteinuria. The product “was also well tolerated, with a safety profile as expected for a locally acting oral budesonide product,” conclude the investigators.
Study participants were 18 years of age or older with eGFRs of 35–90 mL/min/1.73 m2 and persistent proteinuria while on optimized renin-angiotensin system blockade. Randomization to targeted-release budesonide 16 mg/day or placebo for 9 months produced these changes in a primary efficacy endpoint of time-weighted average of eGFR over 2 years: “Patients were recruited to the NefIgArd trial between Sept 5, 2018, and Jan 20, 2021, with 364 patients (182 per treatment group) randomly assigned in the full analysis set. 240 (66%) patients were men and 124 (34%) were women, and 275 (76%) identified as White. The time-weighted average of eGFR over 2 years showed a statistically significant treatment benefit with Nefecon versus placebo (difference 5.05 mL/min per 1.73 m2 [95% CI 3.24 to 7.38], P <0.0001), with a time-weighted average change of –2.47 mL/min per 1.73 m2 (95% CI –3.88 to –1.02) reported with Nefecon and –7.52 mL/min per 1.73 m2 (–8.83 to –6.18) reported with placebo. The most commonly reported treatment-emergent adverse events during treatment with Nefecon were peripheral oedema (31 [17%] patients,vs placebo, seven [4%] patients), hypertension (22 [12%] vs six [3%]), muscle spasms (22 [12%] vs seven [4%]), acne (20 [11%] vs two [1%]), and headache (19 [10%] vs 14 [8%]). No treatment-related deaths were reported.”