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Active, not recruiting Phase 3

Long Term Extension Study in Patients With Primary Hyperoxaluria

NCT04042402 · tracked via the Priya Life Science Australia tracker
Phase
Phase 3
Started
2019-07-09
Last updated
2025-05-30

Condition(s) studied

Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney DiseasesUrologic DiseasesGenetic DiseasePrimary Hyperoxaluria Type 3 (PH3)

Investigational drug(s) / intervention(s)

DCR-PHXC →

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

Study summary

The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (\<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Key Inclusion Criteria: •Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH. * For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 75 days from the last dose of study intervention. * Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged ≥ 18 years, or the multivariate equation by Schwartz in participants aged 12 months to 17 years. In Japan, the cystatin C-based Uemura formula will be used for participants aged 12 months to \<2 years, the creatinine-based Uemura formula by will be used for participants aged 2 to 17 years, and the equation by Matsuo will be used in participants aged ≥ 18 years. Key Exclusion Criteria: * Renal or hepatic transplantation (prior or planned within the study period) * Plasma oxalate \> 30 µmol/L * Currently on dialysis * Documented evidence of clinical manifestations of systemic oxalosis

Primary outcome measure(s)

  • The annual rate of decline in eGFR in participants with PH1 — Annual change from baseline
    To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1

Trial sites (23)

FacilityCityRegionStatus
Clinical Research Site San Francisco California
Clinical Trial Site Boston Massachusetts
Clinical Trial Site Rochester Minnesota
Clinical Trial Site New York New York
Clinical Research Site Herston Queensland
Clinical Trial Site Melbourne Australia
Clinical Research Site Hamilton Ontario
Clinical Trial Site Bron France
Clinical Trial Site Paris France
Clinical Trial Site Bonn Germany
Clinical Trial Site Heidelberg Germany
Clinical Research Site Roma Italy
Clinical Trial Site Fukuoka Japan
Clinical Trial Site Nagoya Japan
Clinical Trial Site Tokyo Japan
Clinical Trial Site Beirut Lebanon
Clinical Trial Site Amsterdam Netherlands
Clinical Trial Site Tromsø Norway
Clinical Research Site Barcelona Spain
Clinical Trial Site Barcelona Spain
Clinical Trial Site Ankara Turkey (Türkiye)
Clinical Trial Site Hampstead London
Clinical Trial Site Birmingham United Kingdom
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04042402 on ClinicalTrials.gov ↗ ← All trials in Australia