Long-Term Safety and Efficacy Study of Nedosiran Injection for Patients with Primary Hyperoxaluria

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What is this study about?

This clinical trial is focused on studying a rare condition called Primary Hyperoxaluria. This is a genetic disorder where the body produces too much oxalate, a substance that can lead to kidney stones and other serious kidney problems. The treatment being tested in this study is a medication called DCR-PHXC, which is given as a solution for injection under the skin. The purpose of the study is to evaluate the long-term safety and effectiveness of this treatment in patients with Primary Hyperoxaluria.

Participants in the study will receive regular injections of DCR-PHXC and will be monitored over time to see how the treatment affects their kidney function. This will be measured by looking at the estimated glomerular filtration rate, or eGFR, which is a test that shows how well the kidneys are working. The study will also track any side effects or changes in health that occur during the treatment period.

The study aims to provide valuable information on how DCR-PHXC can help manage Primary Hyperoxaluria and improve kidney health over the long term. Participants will be closely monitored by healthcare professionals throughout the study to ensure their safety and well-being. This research could lead to better treatment options for those affected by this challenging condition.

1 joining the study

Upon joining the study, the participant will begin a long-term evaluation of the safety and effectiveness of the DCR-PHXC solution for injection. This study is specifically for individuals with a condition known as Primary Hyperoxaluria.

2 medication administration

The medication, nedosiran, is administered as a solution for injection. It is given through a method called subcutaneous use, which means it is injected under the skin.

The frequency and dosage of the medication will be determined by the study protocol and the healthcare team overseeing the trial.

3 monitoring and assessments

Participants will undergo regular monitoring to assess the effect of the medication on kidney function, specifically looking at the estimated glomerular filtration rate (eGFR).

Additional assessments will include physical examinations, vital signs, and laboratory tests such as blood and urine analysis.

4 evaluation of outcomes

The primary outcome of the study is to measure the annual rate of decline in eGFR in participants with PH1.

Secondary outcomes include the incidence and severity of any adverse events, changes in heart activity as measured by an ECG, and changes in the number of kidney stone events.

5 long-term follow-up

The study is designed to continue until March 28, 2031, allowing for long-term follow-up of participants to ensure comprehensive data collection on the safety and efficacy of the treatment.

Who Can Join the Study?

Participants can join the study starting from birth.
Must have a documented diagnosis of Primary Hyperoxaluria (PH), confirmed by a genetic test. Previous genetic information is acceptable.
Must have completed a previous study of the drug DCR-PHXC or be a sibling of someone who completed such a study.
For siblings, they must be younger than 18 years old and meet all other criteria, including genetic testing.
Siblings must have specific urine test results that meet the study’s requirements.
Participants must have a stable urine test result, with less than 20% variation between two tests.
Must have an estimated glomerular filtration rate (eGFR) of at least 30 mL/min, which is a measure of kidney function.
For infants under 12 months, their serum creatinine (a waste product in the blood) must be below a certain level.
Pediatric siblings must weigh at least 12.75 kg.
Male participants with female partners who can become pregnant must agree to use birth control during the study and for 12 weeks after.
Female participants must not be pregnant or breastfeeding and must agree to use birth control if they can become pregnant.
Participants or their guardians must be able to give informed consent, agreeing to follow the study’s rules.
Adolescents aged 12 to under 18 must provide written agreement to participate.
In the Netherlands, children aged 0 to 5 must have PH1 to join the study. Children with PH2 or PH3 cannot join.

Who Cannot Join the Study?

Participants who have a different condition than Primary Hyperoxaluria cannot join. This is a rare condition where the body produces too much of a substance called oxalate.
Participants who are not within the specified age range cannot join. The study is open to certain age groups only.
Participants who are not part of the specified clinical trial groups cannot join. The study is designed for specific groups of people.
Participants who are not male or female cannot join. The study includes both males and females.
Participants who are not considered part of a vulnerable population cannot join. Vulnerable populations may include groups like children or those with certain health conditions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Robert Debre University Hospital	Paris	France
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Hospital Universitario De Canarias	La Laguna	Spain
Centre Hospitalier Lyon Sud	Pierre Benite	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Kvtmfnoxjutyajzygia Bwdv	Bonn	Germany
Ajnirfsvk Uvw	Amsterdam	The Netherlands
Hynatojb Vlth dlgkydzl	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.02.2019
Germany	Not recruiting	01.02.2019
Italy	Not recruiting	01.02.2019
Norway	Not recruiting	01.02.2019
Spain	Not recruiting	01.02.2019
The Netherlands	Not recruiting	01.02.2019

Trial locations

Investigated drugs:

Nedosiran

DCR-PHXC is a solution for injection used in this clinical trial. It is designed to help patients with a condition called Primary Hyperoxaluria, which affects the kidneys. The main goal of using this medication in the trial is to see how it impacts kidney function, specifically by measuring something called the estimated glomerular filtration rate (eGFR). This helps doctors understand how well the kidneys are working in patients receiving this treatment.

Primary Hyperoxaluria – Primary Hyperoxaluria is a rare genetic disorder characterized by the overproduction of oxalate, a substance that can form kidney stones. This condition occurs due to a deficiency in specific liver enzymes, leading to excessive oxalate in the urine. Over time, the high levels of oxalate can cause kidney stones and damage the kidneys. As the disease progresses, it may lead to kidney failure if not managed properly. The condition is typically diagnosed in childhood or early adulthood. It is important to monitor kidney function and manage oxalate levels to prevent complications.

Trial ID:

2024-512260-54-00

Protocol code:

DCR-PHXC-301

NCT ID:

NCT04042402

Trial Phase:

Therapeutic confirmatory (Phase III)

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Long-Term Safety and Efficacy Study of Nedosiran Injection for Patients with Primary Hyperoxaluria

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