A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

Phase 1

Terminated

• Conditions: Primary Hyperoxaluria Type 1
• Interventions: Drug: DCR-PH1; Other: Placebo

• 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

Study Timeline

• Study Start: 2016-05-13
• Study First Submitted: 2016-05-18
• Study First Submitted QC: 2016-06-06
• Study First Posted: 2016-06-10
• Primary Completion: 2016-10-14
• Study Completion: 2016-10-14
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-10
• Last Update Posted: 2024-07-11

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