Long Term Extension Study in Patients With Primary Hyperoxaluria (NCT04042402) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Long Term Extension Study in Patients With Primary Hyperoxaluria
United States, Australia75 participantsStarted 2019-07-09
Plain-language 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.
Who can participate
SexALL
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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
What they're measuring
1
The annual rate of decline in eGFR in participants with PH1
Timeframe: Annual change from baseline
Trial details
NCT IDNCT04042402
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company