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

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

14 criteria

  • 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 trial?

5 criteria

  • 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.
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Investigated drugs

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.

What is already known about the treatment

DCR-PHXC – This medication is administered as a solution for injection, specifically through subcutaneous use. It is currently being studied in clinical trials to assess its long-term safety and effectiveness for treating Primary Hyperoxaluria, a rare genetic condition. The main therapeutic indication for DCR-PHXC is to improve kidney function, as measured by the estimated glomerular filtration rate (eGFR), in patients with this condition. At the molecular level, DCR-PHXC works by targeting and reducing the production of oxalate, a substance that can accumulate and cause kidney damage. It is classified pharmacologically as an RNA interference therapeutic, which means it uses small RNA molecules to interfere with the expression of specific genes.

Investigated diseases

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512260-54-00Protocol codeDCR-PHXC-301Estimated enrolment75 patientsSponsorDicerna Pharmaceuticals Inc.

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