Status:
COMPLETED
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
Lead Sponsor:
Novo Nordisk A/S
Conditions:
Primary Hyperoxaluria Type 1 (PH1)
Primary Hyperoxaluria Type 2 (PH2)
Eligibility:
All Genders
6+ years
Phase:
PHASE2
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
Eligibility Criteria
Inclusion
- Key
- Capable and willing to provide written informed consent or assent
- Documented diagnosis of PH1 or PH2, confirmed by genotyping
- Must meet the 24 hour urine oxalate excretion requirements
- Less than 20% variation between the two 24-hour urinary creatinine excretion values derived from the two 24-hour urine collections in the screening period
- Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA
- Key
Exclusion
- Renal or hepatic transplantation (prior or planned within the study period)
- Currently on dialysis or anticipated requirement for dialysis during the study period
- Plasma oxalate \>30 µmol/L
- 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)
- Use of an RNA interference (RNAi) drug within the last 6 months
- Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening
- Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \>1.5 times upper limit of normal (ULN) for age and gender
- Inability or unwillingness to comply with study procedures
Key Trial Info
Start Date :
October 28 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 29 2021
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT03847909
Start Date
October 28 2019
End Date
June 29 2021
Last Update
May 22 2024
Active Locations (28)
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1
Clinical Trial 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