Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients (NCT04555486) | Clinical Trial Compass
CompletedPhase 1
Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients
United States, Germany, Netherlands6 participantsStarted 2020-09-14
Plain-language summary
The DCR-PHXC-104 study is designed to assess the safety, tolerability, and pharmacological parameters of a single dose of DCR-PHXC in Primary Hyperoxaluria Type 3 (PH3). Participants should have had at least one stone event within 12 months of screening and intact renal function.
Who can participate
Age range6 Years
SexALL
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Key Inclusion Criteria:
* Genetically confirmed PH3
* 24-hour Uox excretion ≥ 0.7 mmol (adjusted per 1.73 m\^2 body surface area \[BSA\] in participants \< 18 years of age) on both assessments conducted in the screening period
* Less than 20% variation between the two 24-hour urinary creatinine excretion values (mmol/kg/24 hours) in the screening period
* Estimated glomerular filtration rate (eGFR) at screening ≥ 30 mL/min, normalized to 1.73 m\^2 BSA
* History of at least one stone event within the last 12 months. Stone events are defined as any of the following:
* renal stone requiring medical intervention, e.g., outpatient procedures such as lithotripsy, or hospitalization or inpatient surgical intervention for confirmed stone-related pain and/or complications;
* stone passage with or without hematuria; or
* renal colic requiring medication.
Key Exclusion Criteria:
* 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)
* Plasma oxalate \> 30 μmol/L
What they're measuring
1
Safety profile of a single dose of DCR-PHXC in PH3 Patients
Timeframe: Screening through Day 85
Trial details
NCT IDNCT04555486
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company