Primary Hyperoxaluria
Conditions
Interventions
investigational material(s)
Generic name etc : Nedosiran, also known as DCR-PHXC
INN of investigational material : -
Therapeutic category code : 399 Agents affecting metabolism, n.e.c.
Dosage and Admi
Sponsors
Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: - 24-hour Uox excretion >= 0.7 mmol (adjusted per 1.73 m2 body surface area [BSA] in participants = 1.6 mmol (adjusted per 1.73 m2 BSA in participants aged = 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.
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy The proportion of participants with a reduction from baseline in 24-hour Uox of at least 70%, based on an AUC and/or reaching normalization or near-normalization of 24-hour Uox on at least 2 consecutive visits, starting from Day 90. Normalization of Uox is defined as = 0.46 to < 0.60 mmol/24 hours (values adjusted per 1.73 m2 BSA in participants aged < 18 years). | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy pharmacokinetics Key Secondary endpoint: AUC from Day 90 to Day 180, based on percent change from Baseline in 24-hour Uox Secondary endpoints: 1. Percent change in the summed surface area and number of kidney stones identified via kidney ultrasound from Baseline to Day 180 2. Percent change in plasma oxalate from Baseline to Day 180 (for adults only) 3. Rate of change in eGFR from Baseline to Day 180 4. AE and SAE; change from Baseline in 12-lead ECG, physical examination findings, vital signs, and clinical laboratory tests 5. Population and individual PK parameters for DCR-PHXC and its metabolites | — |
Countries
Asia except Japan, Japan, North America, Oceania
Outcome results
None listed