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

• Status: Completed
• Trial end date: 2021-06-29
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.

Criteria

Key Inclusion Criteria:

• Capable and willing to provide written informed consent or assent

• Documented diagnosis of PH1 or PH2, confirmed by genotyping

• Must meet the 24 hour urine oxalate excretion requirements

• Less than 20% variation between the two 24-hour urinary creatinine excretion values derived from the two 24-hour urine collections in the screening period

• Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA

Key Exclusion Criteria:

• Renal or hepatic transplantation (prior or planned within the study period)

• Currently on dialysis or anticipated requirement for dialysis during the study period

• Plasma oxalate >30 µmol/L

• Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)

• Use of an RNA interference (RNAi) drug within the last 6 months

• Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening

• 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

• Inability or unwillingness to comply with study procedures