A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)

Phase 2

Completed

• Conditions: Primary Hyperoxaluria

• Registration Number: JPRN-jRCT2080225181
• Lead Sponsor: Dicerna Pharmaceuticals, Inc., Inc. (ICCC: intellim Corporation)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-04-28
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

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.

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified