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Active (not recruiting) Phase 3 NCT04042402

Long Term Extension Study in Patients With Primary Hyperoxaluria

Phase 3 – large-scale trial before approval
Conditions: Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 2 (PH2) Kidney Diseases Urologic Diseases Genetic Disease Primary Hyperoxaluria Type 3 (PH3)

Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

trial.available_in: БГ
Overview
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.
Who can participate
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
Locations 13
Australia (1)
Clinical Research Site
Herston , Queensland
Canada (1)
Clinical Research Site
Hamilton , Ontario
France (1)
Clinical Trial Site
Bron
Germany (1)
Clinical Trial Site
Bonn
Italy (1)
Clinical Research Site
Roma
Japan (1)
Clinical Trial Site
Fukuoka
Lebanon (1)
Clinical Trial Site
Beirut
Netherlands (1)
Clinical Trial Site
Amsterdam
Norway (1)
Clinical Trial Site
Tromsø
Spain (1)
Clinical Research Site
Barcelona
Turkey (1)
Clinical Trial Site
Ankara
United Kingdom (1)
Clinical Trial Site
Hampstead , London
United States (1)
Clinical Research Site
San Francisco , California
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Healthy volunteers
No
Start date
09.07.2019
Completion date
01.04.2030
Registry ID
NCT04042402
Source
anzctr
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Information is automatically extracted from ClinicalTrials.gov. Consult your doctor before taking action.