Overview

This trial is active, not recruiting.

Condition primary hyperoxaluria type 1
Treatment dcr-ph1
Phase phase 1
Sponsor Dicerna Pharmaceuticals, Inc.
Start date May 2016
End date May 2017
Trial size 30 participants
Trial identifier NCT02795325, DCR-PH1-101

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)

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
dcr-ph1
IV infusion of DCR-PH1

Primary Outcomes

Measure
The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs)
time frame: Through Day 29

Secondary Outcomes

Measure
Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax
time frame: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - tmax
time frame: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - AUC
time frame: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - t½
time frame: Through Day 29
The effect of DCR-PH1 on plasma glycolate levels
time frame: Through Day 29
The effect of DCR-PH1 on urine glycolate levels
time frame: Through Day 29
The effect of DCR-PH1 on plasma oxalate levels
time frame: Through Day 29
The effect of DCR-PH1 on urine oxalate levels
time frame: Through Day 29

Eligibility Criteria

Male or female participants at least 12 years old.

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

Additional Information

Official title A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Dicerna Pharmaceuticals, Inc..