What side effects are associated with this type of intervention?

Common treatments for Primary Hyperoxaluria include RNA interference therapeutics like DCR-PHXC, which aim to reduce oxalate production. Known side effects of such treatments can include injection site reactions, flu-like symptoms, and potential liver enzyme abnormalities. Broader treatments for Primary Hyperoxaluria, such as pyridoxine (vitamin B6) and high fluid intake, generally have fewer side effects but may include gastrointestinal discomfort and the challenge of maintaining high fluid intake. In severe cases, dialysis or transplantation may be required, which come with their own risks and complications.

What prior FDA approvals exist?

As of now, there are no specific mentions of prior FDA approvals for treatments of Primary Hyperoxaluria that are similar to DCR-PHXC, an RNA interference therapeutic aimed at reducing oxalate production. The context provided does not include detailed information on previously approved drugs for this condition. Therefore, it is advisable to consult with a healthcare provider for the most current and comprehensive treatment options available.

What other types of research exist?

Promising novel alternatives to DCR-PHXC for Primary Hyperoxaluria patients include gene therapies targeting the underlying genetic mutations, enzyme replacement therapies that provide functional enzymes to reduce oxalate production, and other RNA interference therapies that may offer improved efficacy or safety profiles. Additionally, advancements in small molecule inhibitors that target specific pathways involved in oxalate synthesis are also being explored.

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RNAi Therapeutics

Long-Term DCR-PHXC Treatment for Primary Hyperoxaluria (PHYOX3 Trial)

Phase 3

Waitlist Available

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 ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using appropriate formula based on age and region

Must not have

Documented evidence of clinical manifestations of systemic oxalosis

Currently on dialysis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up monthly for 4 months (d90 through d180)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial aims to provide ongoing access to the treatment DCR-PHXC for patients who were part of earlier studies and their younger siblings. It will also check if the treatment is safe and effective over an extended period. The treatment is intended for people with a condition called Primary Hyperoxaluria (PH). DCR-PHXC is designed to inhibit the enzyme responsible for the final step of oxalate production.

Who is the study for?

This trial is for patients with a genetic kidney condition called Primary Hyperoxaluria who completed previous DCR-PHXC studies, and their siblings under 18 with the same condition. Participants must have a certain level of kidney function and not be on dialysis or have had a kidney or liver transplant.

What is being tested?

The study provides long-term access to DCR-PHXC for those previously enrolled in early-phase trials. It aims to assess the extended safety and effectiveness of this drug in treating Primary Hyperoxaluria.

What are the potential side effects?

While specific side effects are not listed here, they may include typical reactions to medication such as injection site discomfort, potential liver or kidney-related issues due to the nature of the disease being treated, and other common drug-related adverse events.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function is adequate based on a test result.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have symptoms of systemic oxalosis.

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I am currently receiving dialysis.

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I have had or am planning to have a kidney or liver transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ monthly for 4 months (d90 through d180)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~monthly for 4 months (d90 through d180)

This trial's timeline: 3 weeks for screening, Varies for treatment, and monthly for 4 months (d90 through d180) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The annual rate of decline in eGFR in participants with PH1

Secondary study objectives

Change from Baseline in the EQ-5D-5L™ in adults in PH1, PH2, and PH3 participant subgroups

Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children in PH1, PH2, and PH3 participant subgroups

Philadelphia Chromosome

+13 more

Other study objectives

To characterize the PK of DCR PHXC in patients with PH by observing clearance.

To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax).

To characterize the PK of DCR PHXC in patients with PH by observing maximum observed concentration (Cmax).

+5 more

Side effects data

From 2021 Phase 2 trial • 35 Patients • NCT03847909

22%

Injection site erythema

17%

Nausea

17%

Headache

Dysmenorrhoea

Neutrophil count decreased

Injection site pain

Oropharyngeal pain

Nephrolithiasis

Pain in extremity

Abdominal pain

Tachycardia

Fatigue

Blood creatine phosphokinase increased

Arthralgia

Neck pain

Renal colic

Upper respiratory tract infection

100%

80%

60%

40%

20%

Study treatment Arm

DCR-PHXC

Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention

Open label, monthly subcutaneous injection

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

DCR-PHXC

2019

Completed Phase 2

~90

0 / 4Eligibility criteria met

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 is a genetic disorder characterized by the overproduction of oxalate, leading to kidney stones and renal failure. The most common treatments aim to reduce oxalate production or promote its excretion. DCR-PHXC, an RNA interference therapeutic, works by silencing specific genes involved in oxalate production, thereby reducing its levels in the body. This is crucial for patients as it directly addresses the root cause of the disease, potentially preventing kidney damage and improving quality of life. Other treatments may include high fluid intake, dietary modifications, and medications to inhibit oxalate synthesis or promote its excretion.

Molecular mechanism of an adverse drug-drug interaction of allopurinol and furosemide in gout treatment.Deletion of multispecific organic anion transporter Oat1/Slc22a6 protects against mercury-induced kidney injury.