Clinical trial

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

Name

DCR-PHXC-201

Description

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)

Trial arms

Trial start

2019-10-28

Estimated PCD

2021-06-21

Trial end

2021-06-29

Status

Completed

Phase

Early phase I

Treatment

DCR-PHXC

Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection

Arms:

DCR-PHXC

Other names:

nedosiran

Sterile Normal Saline (0.9% NaCl)

Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo

Arms:

Placebo - Sterile Normal Saline (0.9% NaCl)

Size

Primary endpoint

AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox

From Day 90 to 180

Eligibility 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 35, 'type': 'ACTUAL'}}

Updated at

2024-05-22

1 organization

2 products

5 indications

Organization

Novo Nordisk

Product

DCR-PHXC

Indication

Primary Hyperoxaluria Type 1

Indication