Primary Hyperoxaluria Type 1
Conditions
Keywords
Primary Hyperoxaluria Type 1, PH1, Calcium Oxalate Stones, Kidney Stones, Liver Enzyme Deficiency, Genetic Diseases, Inborn Kidney Diseases, Metabolic Diseases, Inborn Errors, Urological Diseases, Carbohydrate Metabolism, Hyperoxaluria, AGT, RNAi, siRNA, DCR-PH1
Brief summary
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
Interventions
DRUGDCR-PH1
IV infusion of DCR-PH1
OTHERPlacebo
Sponsors
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female, at least 12 years of age * Diagnosis of PH1, confirmed by genotyping * 24-hour urine oxalate excretion as defined in the protocol * eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA * Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to \<18 years old, or per local regulatory requirement)
Exclusion criteria
* Prior renal and/or hepatic transplantation * Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment * Pregnancy or lactation at the time of screening or enrollment * Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception * Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus * Moderate to severe liver impairment * Liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) \> 2 times upper limit of normal (ULN) * History of severe reaction to a liposomal product or a known hypersensitivity to lipid products. * Unable to collect required study samples or follow study procedures * No clinically significant health concerns
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs) | Through Day 29 |
Secondary
| Measure | Time frame |
|---|---|
| Profile of pharmacokinetics (PK) of DCR-PH1 - tmax | Through Day 29 |
| Profile of pharmacokinetics (PK) of DCR-PH1 - AUC | Through Day 29 |
| Profile of pharmacokinetics (PK) of DCR-PH1 - t½ | Through Day 29 |
| Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax | Through Day 29 |
| The effect of DCR-PH1 on urine glycolate levels | Through Day 29 |
| The effect of DCR-PH1 on plasma oxalate levels | Through Day 29 |
| The effect of DCR-PH1 on urine oxalate levels | Through Day 29 |
| The effect of DCR-PH1 on plasma glycolate levels | Through Day 29 |
Countries
Germany, Netherlands
Outcome results
None listed