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

July 10, 2024 updated by: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

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

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)

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

41

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Healthy Volunteers
Other: Placebo
Drug: DCR-PH1
IV infusion of DCR-PH1
Experimental: PH Patients
Drug: DCR-PH1
IV infusion of DCR-PH1

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 13, 2016

Primary Completion (Actual)

October 14, 2016

Study Completion (Actual)

October 14, 2016

Study Registration Dates

First Submitted

May 18, 2016

First Submitted That Met QC Criteria

June 6, 2016

First Posted (Estimated)

June 10, 2016

Study Record Updates

Last Update Posted (Actual)

July 11, 2024

Last Update Submitted That Met QC Criteria

July 10, 2024

Last Verified

July 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DCR-PH1-101

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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