Status:
ACTIVE_NOT_RECRUITING
Long Term Extension Study in Patients With Primary Hyperoxaluria
Lead Sponsor:
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Conditions:
Primary Hyperoxaluria Type 1 (PH1)
Primary Hyperoxaluria Type 2 (PH2)
Eligibility:
All Genders
Phase:
PHASE3
Brief 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-t...
Eligibility Criteria
Inclusion
- Key
- •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
- 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
Key Trial Info
Start Date :
July 9 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2030
Estimated Enrollment :
75 Patients enrolled
Trial Details
Trial ID
NCT04042402
Start Date
July 9 2019
End Date
April 1 2030
Last Update
May 30 2025
Active Locations (23)
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1
Clinical Research Site
San Francisco, California, United States, 94143
2
Clinical Trial Site
Boston, Massachusetts, United States, 02115
3
Clinical Trial Site
Rochester, Minnesota, United States, 55905
4
Clinical Trial Site
New York, New York, United States, 10016