Primary hyperoxaluria – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:00 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Primary hyperoxaluria – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of nedosiran for patients with primary hyperoxaluria type 1 and severe kidney impairment with or without dialysis https://clinicaltrials.eu/trial/study-of-nedosiran-for-patients-with-primary-hyperoxaluria-type-1-and-severe-kidney-impairment-with-or-without-dialysis/ Wed, 29 Apr 2026 15:08:34 +0000 https://clinicaltrials.eu/trial/study-of-nedosiran-for-patients-with-primary-hyperoxaluria-type-1-and-severe-kidney-impairment-with-or-without-dialysis/ This study involves patients with Primary Hyperoxaluria Type 1, a rare genetic condition where the liver produces too much of a substance called oxalate. When oxalate builds up in the body, it can combine with calcium to form crystals that damage the kidneys and other organs. The study focuses on patients who have severe kidney damage from this disease, including those who may need dialysis, which is a treatment that uses a machine to filter waste from the blood when the kidneys can no longer do this properly. The treatment being tested is called DCR-PHXC, also known as nedosiran, which is given as an injection under the skin. This medication is designed to reduce the amount of oxalate that the liver produces.

The purpose of this study is to see how well DCR-PHXC works in lowering the levels of oxalate in the blood and to check how safe it is for patients with Primary Hyperoxaluria Type 1 who have severe kidney problems. The study will measure oxalate levels in the blood at the beginning and then again after 180 days of treatment to see if there is a reduction. The study will also look at whether there are changes in the dialysis schedule that patients need, changes in kidney stones that can be seen on ultrasound scans, which use sound waves to create pictures of the inside of the body, and changes in calcium deposits in the kidneys and heart that occur when oxalate crystals build up in these organs.

During the study, patients will receive regular doses of the medication for up to 60 weeks, with a maximum daily dose of 170 milligrams. The study is open to people of different ages, from newborns to adults, and all participants will receive the active treatment rather than a placebo. Throughout the study period, doctors will monitor how well the treatment works and check for any side effects. The study will continue until the end of 2031 to gather long-term information about the safety and effectiveness of this treatment for patients with this rare kidney disease.

]]> Study on the Safety and Effects of ABO-101 with mRNA-002 and gRNA-001 for Patients with Primary Hyperoxaluria Type 1 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-abo-101-with-mrna-002-and-grna-001-for-patients-with-primary-hyperoxaluria-type-1/ Wed, 29 Apr 2026 15:04:57 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-abo-101-with-mrna-002-and-grna-001-for-patients-with-primary-hyperoxaluria-type-1/ This clinical trial is focused on studying a rare genetic condition called Primary Hyperoxaluria Type 1 (PH1). PH1 is a disorder that leads to the overproduction of a substance called oxalate, which can cause kidney stones and other kidney-related issues. The trial will investigate a new treatment known as ABO-101, which is a solution for infusion. This treatment uses advanced gene editing technology to target and disrupt a specific gene called the Hydroxyacid oxidase 1 (HAO1) gene. By doing so, it aims to reduce the activity of an enzyme called glycolate oxidase in liver cells, which in turn decreases the production of oxalate.

The purpose of the study is to evaluate the safety and tolerability of ABO-101 in individuals with PH1. Participants in the study will receive ABO-101 through an intravenous infusion, which means the solution will be delivered directly into the bloodstream. The study will monitor participants over time to assess any side effects and to see how the body processes the treatment. Additionally, the study will measure changes in the levels of oxalate in the urine and other related substances in the blood to understand the treatment’s impact.

This trial is an important step in exploring new treatment options for people with PH1, as current treatments are limited. By participating in this study, researchers hope to gather valuable information that could lead to better management of the condition in the future. The study will involve a series of visits and assessments to ensure the safety and well-being of all participants throughout the trial period.

]]> Study on Stiripentol for Patients Aged 6 and Older with Primary Hyperoxaluria Types 1, 2, or 3 https://clinicaltrials.eu/trial/study-on-stiripentol-for-patients-aged-6-and-older-with-primary-hyperoxaluria-types-1-2-or-3/ Wed, 29 Apr 2026 14:29:23 +0000 https://clinicaltrials.eu/trial/study-on-stiripentol-for-patients-aged-6-and-older-with-primary-hyperoxaluria-types-1-2-or-3/ This clinical trial is focused on studying a rare condition called Primary Hyperoxaluria, which has three subtypes: PH1, PH2, and PH3. This condition causes the body to produce too much of a substance called oxalate, which can lead to kidney stones and other health issues. The trial will evaluate the effectiveness and safety of a medication called stiripentol, which is taken in the form of hard capsules. Stiripentol is being tested to see if it can help reduce the amount of oxalate in the urine of patients who are 6 years and older.

The purpose of the study is to determine if stiripentol can decrease urinary oxalate excretion, which is the amount of oxalate that is passed out of the body through urine. Participants in the study will receive either stiripentol or a placebo, which looks like the medication but does not contain the active ingredient. The study will last for a period of up to 60 days, during which participants will take the medication orally, meaning by mouth. The trial will monitor changes in the amount of oxalate in the urine over time, as well as any changes in kidney function and the occurrence of kidney stones.

Throughout the study, participants will be regularly assessed to track their health and any side effects they may experience. The trial aims to provide valuable information on whether stiripentol can be a beneficial treatment for those living with Primary Hyperoxaluria. This research could potentially lead to new treatment options for managing this challenging condition.

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