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

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 - DCR-PHXC-201 (PHYOX 2)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON49278

Enrollment

Registered

2019-02-07

Start date

2020-08-26

Completion date

Unknown

Last updated

2024-04-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hyperoxaluria Primary Hyperoxaluria

Interventions

DCR-PHXC is a synthetic ribonucleic acid interference (RNAi) drug that consists of double-stranded oligonucleotides conjugated to a GalNAc ligand. DCR-PHXC is a pale yellow, sterile solution of the

Eligibility

Age

2 Years to 99 Years

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

Exclusion criteria

Exclusion criteria: Key exclusion criteria: - Prior renal or hepatic transplantation; or planned transplantation within the study period - Currently receiving dialysis or anticipating requirement for dialysis during the study period - Plasma oxalate > 30 *mol/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). - 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
Primary End point: The proportion of participants with a reduction from baseline in 24-hour Uox of at least 70%, based on a 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	—

Secondary

Measure	Time frame
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 Exploratory Endpoints 1. Number of stone events over a 6-month period 2. TWS AUC of 24-hour Uox from Day 1 to Day 180, based on percent change from Baseline 3. Percent change in 24-hour Uox from Baseline to Day 180 4. TWS AUC of 24-hour urinary oxalate-to-creatinine ratio from Day 90 to Day 180, based on percent change from Baseline 5. Change from Baseline to Day 180 in the SF-36 and EQ-5D--5L in adults; and in the PedsQL* in children 6. Uox in spot urine and 24-hour urine	—

Measure

Time frame

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 Exploratory Endpoints 1. Number of stone events over a 6-month period 2. TWS AUC of 24-hour Uox from Day 1 to Day 180, based on percent change from Baseline 3. Percent change in 24-hour Uox from Baseline to Day 180 4. TWS AUC of 24-hour urinary oxalate-to-creatinine ratio from Day 90 to Day 180, based on percent change from Baseline 5. Change from Baseline to Day 180 in the SF-36 and EQ-5D--5L in adults; and in the PedsQL* in children 6. Uox in spot urine and 24-hour urine

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)