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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-13

Study Completion Date

2016-10-14

Brief Summary

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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)

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Detailed Description

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Conditions

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Primary Hyperoxaluria Type 1

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PH Patients

Group Type EXPERIMENTAL

DCR-PH1

Intervention Type DRUG

IV infusion of DCR-PH1

Healthy Volunteers

Group Type EXPERIMENTAL

DCR-PH1

Intervention Type DRUG

IV infusion of DCR-PH1

Placebo

Intervention Type OTHER

Interventions

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DCR-PH1

IV infusion of DCR-PH1

Intervention Type DRUG

Placebo

Intervention Type OTHER

Eligibility Criteria

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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

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No