A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria

Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)0 个研究点目标入组 5 人开始时间: 2020年4月28日最近更新: 2023年10月17日

研究概览

暂无简介。

•Key Inclusion Criteria:
•- 24-hour Uox excretion >= 0.7 mmol (adjusted per 1.73 m2 body surface area [BSA] in participants < 18 years of age) in both collections performed in the screening period. Of the first 24 participants enrolled, at least 12 (50%) must have at least one 24-hour Uox excretion >= 1.6 mmol (adjusted per 1.73 m2 BSA in participants aged < 18 years).
•- Less than 20% variation between the two 24-hour urinary creatinine excretion values [mmol/24 hr/kg] derived from the two 24-hour urine collections in the screening period.
•- Estimated glomerular filtration rate (eGFR) at screening >= 30 mL/min normalized to 1.73 m2 BSA calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula in participants aged >= 18 years, or the formula by Schwartz in participants aged 6 to 17 years. In Japan, the formula by Uemura et al. will be used for participants aged 6 to 17 years, and the formula by Matsuo et al. will be used in participants aged >= 18 years.

•Key exclusion criteria include:
•- Renal or hepatic transplantation (prior or planned within the study period)
•- Current dialysis or anticipated requirement for dialysis during the study period
•- Plasma oxalate > 30 micromol/L
•- Documented evidence of clinical manifestations of systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
•- Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 1.5 times upper limit of normal (ULN) for age and gender

发起方

Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)