Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
This study is investigating a new medicine, DCR-PHXC, for individuals living with Primary Hyperoxaluria Type 1 (PH1) and severe kidney disease, including those on dialysis. PH1 is a rare genetic condition that can seriously affect the kidneys. The main goals are to check the medicine's safety and how well it works. This is a Phase 2 study, meaning it's still in the earlier stages of human testing, after initial safety checks. Participants will receive the medicine and be closely monitored by doctors over several years. This research is important because it could lead to new treatment options for people with this challenging condition.
At a glance
What is this study about?
This research study is looking at a new medicine called DCR-PHXC for people who have a rare genetic condition called Primary Hyperoxaluria Type 1, or PH1 for short. People with PH1 produce too much of a substance called oxalate, which can build up and severely damage their kidneys, sometimes leading to end-stage kidney (renal) disease (ESRD). This means their kidneys no longer work well enough to keep them healthy, and they might need dialysis or a kidney transplant.
The study wants to understand if DCR-PHXC is a safe medicine for people with PH1 and severe kidney problems. It also aims to see how well it works. This includes people who might be on dialysis already. Because this is a Phase 2 study, it means that the medicine has already gone through some initial safety tests and researchers are now looking to learn more about its effects in a larger group of people with the condition.
The information gathered from this study is really important. If DCR-PHXC proves to be safe and effective, it could offer a new way to help manage PH1 and its impact on the kidneys, potentially improving the lives of individuals with this challenging condition.
Key takeaways
- Tests a new medicine, DCR-PHXC, for PH1 with severe kidney disease.
- Aims to check safety and how well the medicine works.
- Currently recruiting only children under 6 years old, but plans to expand to all ages.
- Involves monthly visits for 6 months, then longer-term follow-up.
- Could offer a new treatment option for a rare, serious kidney condition.
Who may be eligible?
Currently, this study is only looking for children under 6 years old to participate. However, the plan is to eventually include people of all ages, from newborns to adults, who have PH1.
To be considered, you must have a confirmed diagnosis of PH1 through genetic testing. Your kidneys must also be severely damaged, with a specific measure of kidney function (called GFR) below a certain level. Additionally, a blood test for oxalate levels needs to show that they are higher than usual.
If you are on dialysis (either hemodialysis or peritoneal dialysis), you might be able to join if you haven't been on it for more than two years and your dialysis routine has been stable for at least two weeks before the study starts. Both men and women can participate. If you are a woman who could become pregnant, or a man with a partner who could become pregnant, you will need to agree to use effective birth control during and for some time after the study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do you have a genetically confirmed diagnosis of Primary Hyperoxaluria Type 1 (PH1)?
- Are your kidneys severely damaged (low GFR score)?
- Is your oxalate level in your blood higher than normal?
- If on dialysis, have you been on it for less than 2 years and has it been stable for at least 2 weeks?
- If you are a woman of childbearing potential, are you able to use effective birth control during and after the study?
What does participation involve?
If you decide to take part, you'll first go through a screening period of up to 35 days to make sure you meet all the study requirements. After that, you'll visit the clinic once a month for about six months (180 days) to receive the DCR-PHXC medicine and have your health checked.
If you complete these initial visits successfully, you'll then enter a longer follow-up period, possibly for up to five more years. During this extended period, you'll continue to receive DCR-PHXC. You'll visit the clinic every three months. To make things easier, you or your caregiver might be taught how to give the medicine at home, or a home health nurse could help. The study is expected to last about five years in total from the start to the end of the initial six-month treatment for the last participant, with an additional five years of extended follow-up for those who continue.
Throughout your participation, the study team will monitor your health closely, take blood tests, and assess how the medicine is affecting your PH1 and kidney function.
Potential risks and benefits
Locations (18)
- Clinical Trial SiteVerified postcodeSan Francisco, United States· Active not recruiting
- Clinical Trial SiteVerified postcodeBoston, United States· Active not recruiting
- Clinical Trial SiteVerified postcodeRochester, United States· Recruiting
- Clinical Trial SiteVerified postcodeNew York, United States· Active not recruiting
- Clinical Trial SiteVerified postcodeBron, France· Withdrawn
- Clinical Trial SiteVerified postcodeParis, France· Withdrawn
- Clinical Trial SiteVerified postcodeBonn, Germany· Recruiting
- Clinical Trial SiteVerified postcodeHeidelberg, Germany· Withdrawn
- Clinical Trial SiteVerified postcodeRoma, Italy· Withdrawn
- Clinical Trial SiteVerified postcodeBeirut, Lebanon· Recruiting
- Clinical Trial SiteVerified postcodeCasablanca, Morocco· Withdrawn
- Clinical Trial SiteVerified postcodeOradea, Romania· Withdrawn
Common questions
What is Primary Hyperoxaluria Type 1 (PH1)?
PH1 is a rare inherited condition where your body makes too much oxalate, a substance that can build up and cause kidney stones and serious kidney damage.
What is End Stage Renal Disease (ESRD)?
ESRD means your kidneys have failed and can no longer clean your blood well enough to keep you healthy. People with ESRD often need dialysis or a kidney transplant.
How long does the study last?
The initial part of the study involves monthly visits for about 6 months, followed by an extended follow-up period where you could receive the medicine for up to 5 more years. The total study duration, including all participants, is expected to be around 5 years, plus the extended follow-up.
Will I have to pay to be in the study?
Clinical studies are typically funded by the sponsor (in this case, Novo Nordisk). You will not be charged for the study medicine or related study visits and tests. You might be reimbursed for travel expenses related to your attendence. This will be discussed with you before you agree to participate.
What does 'Phase 2 study' mean?
A Phase 2 study means the medicine has been shown to be safe in a small group of people, and now researchers want to see how well it works and continue checking its safety in a larger group of people who have the condition.
How to find out more
Novo Nordisk
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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