Long Term Extension Study in Patients With Primary Hyperoxaluria
This study is for people who have already taken part in earlier research for a medicine called DCR-PHXC, which treats a rare kidney condition called Primary Hyperoxaluria (PH). It also welcomes their siblings under 18 years old who have PH. The main goal is to let these patients continue getting DCR-PHXC for a longer time. Researchers will closely watch to see if the medicine is safe and if it continues to work well in managing PH. This helps gather more information about the long-term effects of DCR-PHXC, which is important for understanding how best to treat this serious genetic condition affecting the kidneys.
At a glance
What is this study about?
This study is looking at a medicine called DCR-PHXC, which is designed to help people with a rare genetic condition called Primary Hyperoxaluria (PH). PH causes too much of a substance called oxalate to build up in the body, which can severely damage the kidneys and other organs. There are different types of PH, including PH1, PH2, and PH3.
This particular study is a 'long-term extension study.' This means it's for people who have already been part of earlier studies looking into DCR-PHXC. It gives them the chance to keep receiving the treatment. It's also open to younger brothers or sisters (under 18) who have PH. The main purpose is to find out if DCR-PHXC is safe and effective when used over a longer period.
By gathering more information over time, doctors and researchers can better understand how this new treatment works and whether it can safely help patients manage their PH in the long run. This is crucial for developing better treatment options for this challenging kidney disease.
Key takeaways
- This study is for long-term treatment of Primary Hyperoxaluria (PH).
- It's open to previous study participants and their younger siblings with PH.
- The medicine being studied is called DCR-PHXC.
- Researchers will check how safe and effective the medicine is over time.
- Good kidney function is generally required to join.
- You cannot join if you are on dialysis or have had an organ transplant.
Who may be eligible?
This study is mainly for people who have already successfully taken part in a previous study of DCR-PHXC. If you've been in one of those studies, you might be able to join this one.
Also, brothers or sisters under the age of 18 who have been diagnosed with PH through a genetic test may be able to join if their sibling was in a previous DCR-PHXC study.
There are a few health requirements, including having reasonably good kidney function (your doctor can check this). You can't join if you've had a kidney or liver transplant, are currently on dialysis, or have signs that oxalate has severely affected other parts of your body.
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.
- Have you previously taken part in a DCR-PHXC study and completed it?
- If not, do you have a brother or sister who completed a DCR-PHXC study, and are you under 18 with genetically confirmed PH?
- Do you have reasonably good kidney function (your doctor can check this)?
- Have you not had a kidney or liver transplant?
- Are you not currently receiving dialysis treatment?
- Do you not have signs of widespread oxalate damage in your body?
What does participation involve?
If you join this study, you will continue to receive the study medicine, DCR-PHXC. The exact schedule for taking the medicine and for your clinic visits will be explained by the study team. You will have regular appointments where doctors will check your general health, kidney function, and the levels of certain substances in your blood and urine to see how the medicine is working and if it's safe for you. These checks will involve blood tests and urine samples, and possibly other tests as needed. The study aims to follow participants for a longer period, so you'll have ongoing monitoring and follow-up care.
Potential risks and benefits
Locations (23)
- Clinical Research SiteVerified postcodeSan Francisco, United States
- Clinical Trial SiteVerified postcodeBoston, United States
- Clinical Trial SiteVerified postcodeRochester, United States
- Clinical Trial SiteVerified postcodeNew York, United States
- Clinical Research SiteVerified postcodeHerston, Australia
- Clinical Trial SiteVerified postcodeMelbourne, Australia
- Clinical Research SiteVerified postcodeHamilton, Canada
- Clinical Trial SiteVerified postcodeBron, France
- Clinical Trial SiteVerified postcodeParis, France
- Clinical Trial SiteVerified postcodeBonn, Germany
- Clinical Trial SiteVerified postcodeHeidelberg, Germany
- Clinical Research SiteVerified postcodeRoma, Italy
Common questions
What is Primary Hyperoxaluria?
It's a rare genetic condition where the body makes too much of a substance called oxalate, which can build up and damage the kidneys and other organs.
What is DCR-PHXC?
It's the name of the new medicine being studied to help treat Primary Hyperoxaluria.
Who can join this study?
Mostly people who've already been in DCR-PHXC studies, or their siblings under 18 with a confirmed diagnosis of PH.
Will I have to pay to be in the study?
No, all study-related medical care and the study medicine will be provided without charge.
How long will the study last for me?
The study is for long-term treatment, so you will continue to receive the medicine and be monitored as long as it's safe and beneficial, and you wish to continue.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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