DCR-PHXC for Kidney Failure
(PHYOX7 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called DCR-PHXC (also known as Nedosiran) for individuals with a rare genetic condition called PH1, which leads to kidney failure. The treatment involves a monthly injection and targets those with severe kidney problems, regardless of dialysis status. It is suitable for individuals diagnosed with PH1 who experience serious kidney issues affecting daily life. Participants should not have had a liver transplant, though a previous kidney transplant is acceptable. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.
Do I need to stop my current medications to join the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that DCR-PHXC is likely to be safe for humans?
Research has shown that DCR-PHXC, also known as nedosiran, has been studied in patients with a kidney condition called PH1. In these studies, DCR-PHXC was generally well-tolerated, with most patients not experiencing serious side effects. For instance, patients with healthier kidneys did not encounter major problems. The treatment has a good safety record, with severe negative reactions being rare. Some individuals might experience mild or moderate side effects, but these are uncommon and not serious.
The current trial is in phase 2, indicating that DCR-PHXC has already passed initial safety tests, suggesting it is safe enough for testing in more people. However, like any treatment, some risks may remain, and results can vary from person to person.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for kidney failure, which often include dialysis or kidney transplants, DCR-PHXC offers a new approach with its unique mechanism of targeting the root cause of the condition. DCR-PHXC is a subcutaneous injection that utilizes RNA interference technology to specifically reduce the production of oxalate, a key contributor to kidney damage. Researchers are excited about this treatment because it not only targets the disease at a molecular level but also provides a non-invasive administration method compared to traditional procedures. This has the potential to improve patient outcomes and quality of life significantly.
What evidence suggests that DCR-PHXC might be an effective treatment for kidney failure?
Research has shown that DCR-PHXC, also known as nedosiran, may help treat primary hyperoxaluria type 1 (PH1) in patients with serious kidney problems. In this trial, participants will receive an open-label monthly subcutaneous injection of DCR-PHXC. Studies have found that patients with PH1 who took nedosiran experienced a significant drop in urinary oxalate levels. This is important because high oxalate levels can harm the kidneys. A preliminary analysis revealed that patients had an average urinary oxalate level of 0.88 mmol/24h per 1.73 square meters, an important measure of treatment success. Additionally, earlier research showed that nedosiran is well-tolerated and safe for patients with severe kidney issues. Overall, these findings suggest that DCR-PHXC could be a helpful option for those dealing with PH1 and kidney problems.16789
Who Is on the Research Team?
Clinical Transparency (dept. 2834)
Principal Investigator
Novo Nordisk A/S
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive monthly subcutaneous injections of DCR-PHXC based on age and weight
Extended Follow-up
Participants continue to receive open-label DCR-PHXC for an additional 5 years or until commercially available
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- DCR-PHXC
DCR-PHXC is already approved in United States for the following indications:
- Primary hyperoxaluria type 1 (PH1)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Dicerna Pharmaceuticals, Inc.
Lead Sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Lead Sponsor