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RNAi Therapeutics

DCR-PHXC for Kidney Failure (PHYOX7 Trial)

Phase 2
Recruiting
Research Sponsored by Dicerna Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Estimated GFR at Screening <30mL/min normalized to 1.73m^2 BSA
Documented diagnosis of PH1 or PH2, confirmed by genotyping
Must not have
Liver function test abnormalities: ALT and/or AST >1.5 × ULN for age and gender
Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 180 days
Awards & highlights

Summary

This trial is testing a new medication called DCR-PHXC for people with severe kidney disease (PH1 or PH2). The treatment aims to protect the kidneys and improve their function. Participants will receive the medication over a period of time, with an option for continued monitoring.

Who is the study for?
This trial is for individuals with Primary Hyperoxaluria (PH1 or PH2) and severe kidney impairment, who may or may not be on dialysis. Participants must have a certain body weight, provide consent, have health insurance if required by law, and meet specific genetic diagnosis and plasma oxalate levels. There are age-specific groups from newborns to adults. Those on dialysis should have been stable for at least 2 weeks.
What is being tested?
The study tests DCR-PHXC in patients with PH1/2 who also suffer from end-stage renal disease (ESRD). It aims to assess the safety and effectiveness of this intervention in improving their condition regardless of whether they're undergoing dialysis treatment.
What are the potential side effects?
Potential side effects include reactions related to oligonucleotide-based therapies which could involve immune responses like inflammation or sensitivity issues due to ingredients in DCR-PHXC. Specific side effect profiles will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My kidney function is severely reduced.
Select...
My diagnosis of PH1 or PH2 is confirmed by genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My liver tests are higher than normal for my age and gender.
Select...
I have had a liver transplant or will have one soon. Kidney transplants are okay.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~180 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 180 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Safety: Incidence of Events
Secondary study objectives
Change from Baseline in Plasma Oxalate Concentration
Change in Duration of Dialysis
Change in Frequency of Dialysis
+5 more
Other study objectives
Change from Baseline in the EQ-5D-5L™ in adults
Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children
Change from Baseline in the Short Form (36) Health Survey (SF-36®) in adults
+2 more

Side effects data

From 2021 Phase 2 trial • 35 Patients • NCT03847909
22%
Injection site erythema
17%
Nausea
17%
Headache
9%
Dysmenorrhoea
9%
Neutrophil count decreased
9%
Injection site pain
9%
Oropharyngeal pain
9%
Nephrolithiasis
9%
Pain in extremity
9%
Abdominal pain
4%
Tachycardia
4%
Fatigue
4%
Blood creatine phosphokinase increased
4%
Arthralgia
4%
Neck pain
4%
Renal colic
4%
Upper respiratory tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
DCR-PHXC
Placebo

Trial Design

1Treatment groups
Experimental Treatment
Group I: Open-Label DCR-PHXCExperimental Treatment1 Intervention
Open-Label monthly subcutaneous injection of DCR-PHXC based on age and weight.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
DCR-PHXC
2019
Completed Phase 2
~90

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Primary Hyperoxaluria (PH) treatments primarily aim to reduce oxalate production or mitigate its harmful effects. Glycolate oxidase inhibitors, such as CCPST, decrease oxalate production by inhibiting the enzyme that converts glycolate to glyoxylate, a precursor of oxalate. Antioxidants like N-acetylcysteine (NAC) and quercetin help reduce oxidative stress and renal damage caused by high oxalate levels. The DCR-PHXC trial focuses on substrate reduction therapy, targeting metabolic pathways to lower oxalate production. This approach is vital for PH patients as it addresses the disease's root cause, potentially reducing kidney stone formation and preserving renal function.
Protective Effects of Quercetin on Oxidative Stress-Induced Tubular Epithelial Damage in the Experimental Rat Hyperoxaluria Model.Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I.Renal cell adaptation to oxalate.

Find a Location

Who is running the clinical trial?

Dicerna Pharmaceuticals, Inc.Lead Sponsor
14 Previous Clinical Trials
470 Total Patients Enrolled
2 Trials studying Primary Hyperoxaluria
68 Patients Enrolled for Primary Hyperoxaluria
Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyLead Sponsor
18 Previous Clinical Trials
550 Total Patients Enrolled
2 Trials studying Primary Hyperoxaluria
68 Patients Enrolled for Primary Hyperoxaluria
Alexandra Haagensen, MD, MBAStudy ChairDicerna Pharmaceuticals, Inc.
3 Previous Clinical Trials
41 Total Patients Enrolled
1 Trials studying Primary Hyperoxaluria
25 Patients Enrolled for Primary Hyperoxaluria

Media Library

DCR-PHXC (RNAi Therapeutics) Clinical Trial Eligibility Overview. Trial Name: NCT04580420 — Phase 2
Primary Hyperoxaluria Research Study Groups: Open-Label DCR-PHXC
Primary Hyperoxaluria Clinical Trial 2023: DCR-PHXC Highlights & Side Effects. Trial Name: NCT04580420 — Phase 2
DCR-PHXC (RNAi Therapeutics) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04580420 — Phase 2
~0 spots leftby Nov 2024
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