A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
- Registration Number
- NCT02795325
- Lead Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- 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)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 41
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description PH Patients DCR-PH1 - Healthy Volunteers DCR-PH1 - Healthy Volunteers Placebo -
- Primary Outcome Measures
Name Time Method The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs) Through Day 29
- Secondary Outcome Measures
Name Time Method Profile of pharmacokinetics (PK) of DCR-PH1 - t½ Through Day 29 Profile of pharmacokinetics (PK) of DCR-PH1 - AUC Through Day 29 Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax Through Day 29 Profile of pharmacokinetics (PK) of DCR-PH1 - tmax Through Day 29 The effect of DCR-PH1 on plasma 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 urine glycolate levels Through Day 29