Ongoing Clinical Trials for Hyperoxaluria
There are currently 2 clinical trials investigating treatments for hyperoxaluria, a rare genetic condition where the body produces too much oxalate, which can lead to kidney stones and serious kidney problems. These studies are testing injectable medications designed to reduce oxalate levels and protect kidney function. Trials are taking place across several European countries including Belgium, France, Germany, Italy, the Netherlands, and Spain.
Clinical trial locations
- Belgium
- France
- Germany
- Italy
- Netherlands
- Spain
Long-Term Safety and Efficacy Study of Nedosiran Injection for Patients with Primary Hyperoxaluria
This trial is testing a medication called DCR-PHXC (nedosiran), which is injected under the skin to treat patients with Primary Hyperoxaluria. The study will run until March 2031 to evaluate the long-term safety and effectiveness of this treatment.
Main inclusion criteria: Participants can join from birth and must have a genetically confirmed diagnosis of Primary Hyperoxaluria. They must have previously completed another study of DCR-PHXC or be a sibling of someone who did. Kidney function must be adequate, with an estimated glomerular filtration rate (eGFR) of at least 30 mL/min, which measures how well the kidneys filter waste. For infants under 12 months, specific blood creatinine levels apply. Pediatric siblings must weigh at least 12.75 kg. Participants must agree to use birth control if applicable and provide informed consent. In the Netherlands, children aged 0 to 5 can only join if they have PH1, not other types.
Main exclusion criteria: Patients with conditions other than Primary Hyperoxaluria cannot participate. The study is limited to specific age groups and clinical trial groups. Both males and females can participate.
Focus and goals: The trial aims to measure how nedosiran affects the annual rate of decline in kidney function, specifically the eGFR. Researchers will monitor participants’ kidney health through regular physical examinations, vital signs, blood tests, and urine analysis. The study also tracks any side effects, changes in heart activity through ECG, and the number of kidney stone events. The long-term design allows comprehensive data collection on safety and effectiveness.
Investigational drug: DCR-PHXC (nedosiran) is an RNA interference therapeutic that works by reducing the production of oxalate in the body. By targeting specific genes that cause oxalate overproduction, this medication aims to prevent the kidney damage that occurs when oxalate accumulates and forms stones.
Study on Lumasiran for Patients with Advanced Primary Hyperoxaluria Type 1
This trial is studying lumasiran (ALN-GO1), an injectable treatment for patients with advanced Primary Hyperoxaluria Type 1 (PH1). The study includes two groups: patients not on dialysis and patients who are on dialysis. Dialysis is a treatment that helps filter waste from the blood when kidneys aren’t working properly.
Main inclusion criteria: Participants must be full-term infants at the time of consent and have genetically confirmed PH1. Adults aged 18 or older must have an eGFR of 45 mL/min/1.73 m² or less, indicating reduced kidney function. Younger patients have different kidney function requirements based on age. Blood plasma oxalate levels must average 20 micromoles per liter or higher based on three recent tests. If taking vitamin B6 (pyridoxine), patients must have been on a stable dose for at least 90 days and continue it until the 6-month visit. Participants in the dialysis group must have been on a stable hemodialysis schedule for at least 4 weeks before the study. Written informed consent is required, with parental consent for minors.
Main exclusion criteria: Patients without PH1 cannot participate. Those outside the specified age range, unwilling or unable to follow study procedures, with interfering medical conditions, currently in another trial, pregnant, breastfeeding, with allergies to the study medication, or unable to provide informed consent are excluded.
Focus and goals: The study evaluates how effectively lumasiran reduces plasma oxalate levels in the blood over several months. Researchers measure the percent change in plasma oxalate levels from the start of the study to month 6. The trial also assesses kidney function, quality of life, and overall health impacts. Regular follow-up visits include blood tests and health assessments, with additional monitoring between dialysis sessions for patients in that group.
Investigational drug: Lumasiran is a small interfering RNA (siRNA) therapeutic administered as a 94.5 mg/0.5 mL subcutaneous injection. It works by targeting and reducing the production of substances in the liver that lead to oxalate formation. By lowering oxalate levels, lumasiran may help prevent kidney stones and the serious complications that can occur when oxalate deposits accumulate in organs and tissues throughout the body.
Summary
Both trials are investigating innovative RNA-based therapies for Primary Hyperoxaluria, representing significant advances in treating this rare genetic condition. The studies are concentrated in Western European countries, particularly France, Italy, and the Netherlands, which appear in both trials. This geographical concentration likely reflects the presence of specialized medical centers with expertise in rare kidney diseases.
The nedosiran trial takes a broader approach, including all types of Primary Hyperoxaluria and following patients long-term until 2031, while the lumasiran trial focuses specifically on advanced PH1 patients, including those requiring dialysis. Both medications use RNA interference technology to reduce oxalate production at its source in the liver, rather than simply treating symptoms. These trials offer hope for patients with this challenging condition, particularly those with advanced disease where kidney function is already compromised.
