Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-14

Study Completion Date

2021-09-07

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The DCR-PHXC-104 study is designed to assess the safety, tolerability, and pharmacological parameters of a single dose of DCR-PHXC in Primary Hyperoxaluria Type 3 (PH3). Participants should have had at least one stone event within 12 months of screening and intact renal function.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

NCT04580420

Primary Hyperoxaluria Type 1 End Stage Renal Disease

RECRUITING PHASE2

A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2

NCT03847909

Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 2 (PH2) Kidney Diseases +2 more

COMPLETED PHASE2

Long Term Extension Study in Patients With Primary Hyperoxaluria

NCT04042402

Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 2 (PH2) Kidney Diseases +3 more

ACTIVE_NOT_RECRUITING PHASE3

Natural History of Patients With PH3 and a History of Stone Events

NCT04542590

Primary Hyperoxaluria Type 3

COMPLETED

A 4-Week Dose-Ranging and Efficacy Trial of KRX-0502 (Ferric Citrate) in Patients With End-Stage Renal Disease

NCT01074125

Hyperphosphatemia End-Stage Renal Disease

COMPLETED PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Potential participants are screened over an up-to-35-day period (with an extra 7-day period for participants who are required to repeat screening 24-hour urine collections or initially unanalyzable screening laboratory assessment samples) prior to randomization. Eligible participants will receive a single dose of DCR-PHXC or placebo on Day 1.

In order to maintain the treatment blind, 24-hour urine oxalate (Uox) results that could unblind the study will not be reported to investigative sites or other blinded personnel until the study has been unblinded.

It is expected that approximately 10 participants will be screened in order to randomize 6 participants (2:1 randomization; 4 nedosiran:2 placebo) to the study.

Following the up-to-6-week screening period, participants will return to the clinic for interim visits up to Day 85. Visits occurring between the Day 1 and the Day 85 visit may be conducted as at-home telemedicine visits at the discretion of the Investigator. The total time on study for each participant is approximately 18 weeks.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Primary Hyperoxaluria Type 3

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Participants are randomly assigned to one of two interventions: the study drug DCR-PHXC (also known as nedosiran) or the placebo comparator Sterile Normal Saline. For every 2 participants that receive DCR-PHXC, 1 participant will receive placebo. Thus, 4 participants are expected to receive DCR-PHXC, and 2 participants are expected to receive placebo.

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

DCR-PHXC

Participants that are at least 12 years old will receive a single dose of 3 mg/kg DCR-PHXC (or nedosiran) via subcutaneous (SC) injection. Participants that are 6-11 years old will receive a single dose of 3.5 mg/kg DCR-PHXC (nedosiran) via SC injection.

Group Type EXPERIMENTAL

DCR-PHXC

Intervention Type DRUG

Intervention, drug, DCR-PHXC

Sterile Normal Saline (0.9% NaCl)

Participants will receive a single dose of Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo.

Group Type PLACEBO_COMPARATOR

Sterile Normal Saline (0.9% NaCl)

Intervention Type DRUG

Placebo comparator

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

DCR-PHXC

Intervention, drug, DCR-PHXC

Intervention Type DRUG

Sterile Normal Saline (0.9% NaCl)

Placebo comparator

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Nedosiran

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Genetically confirmed PH3
* 24-hour Uox excretion ≥ 0.7 mmol (adjusted per 1.73 m\^2 body surface area \[BSA\] in participants \< 18 years of age) on both assessments conducted in the screening period
* Less than 20% variation between the two 24-hour urinary creatinine excretion values (mmol/kg/24 hours) in the screening period
* Estimated glomerular filtration rate (eGFR) at screening ≥ 30 mL/min, normalized to 1.73 m\^2 BSA
* History of at least one stone event within the last 12 months. Stone events are defined as any of the following:

* renal stone requiring medical intervention, e.g., outpatient procedures such as lithotripsy, or hospitalization or inpatient surgical intervention for confirmed stone-related pain and/or complications;
* stone passage with or without hematuria; or
* renal colic requiring medication.

Exclusion Criteria

* Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
* Plasma oxalate \> 30 μmol/L

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No