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Nedosiran

Nedosiran is an investigational drug currently being studied in clinical trials for the treatment of Primary Hyperoxaluria (PH), particularly Type 1 (PH1). This article explores the ongoing research on Nedosiran, its potential benefits, and its administration in patients of various ages with PH. The trials aim to evaluate the safety, efficacy, and pharmacokinetics of Nedosiran in both adults and children with this rare genetic disorder.

Table of Contents

What is Nedosiran?

Nedosiran, also known as DCR-PHXC, is an investigational drug being developed to treat a rare genetic condition called Primary Hyperoxaluria (PH)[1]. It’s important to understand that as an investigational drug, nedosiran is still being studied and is not yet approved for general use by regulatory agencies.

What Conditions Does Nedosiran Treat?

Nedosiran is specifically designed to treat Primary Hyperoxaluria (PH), a rare genetic disorder that affects the kidneys. There are three main types of PH that nedosiran may potentially treat[2]:

  • Primary Hyperoxaluria Type 1 (PH1): This is the most common and severe form of PH.
  • Primary Hyperoxaluria Type 2 (PH2)
  • Primary Hyperoxaluria Type 3 (PH3)

In Primary Hyperoxaluria, the body produces too much of a substance called oxalate. This excess oxalate can combine with calcium to form kidney stones and can also damage the kidneys over time. Nedosiran aims to reduce the production of oxalate in the body, potentially slowing or preventing kidney damage in people with PH[1][2].

How is Nedosiran Administered?

Nedosiran is given as a subcutaneous injection, which means it’s injected just under the skin. The dosing schedule and amount can vary depending on the patient’s age and weight[1]:

  • For adults and adolescents (aged 12-17 years) weighing 50 kilograms (kg) or more: 170 milligrams (mg) once a month
  • For adults and adolescents weighing less than 50 kg: 136 mg once a month
  • For children aged 6 to 11 years: 3.5 mg per kg of body weight monthly, not exceeding 170 mg

The injection is typically given in the thigh or abdomen. In some cases, after proper training, patients or their caregivers may be able to administer the medication at home[1].

Clinical Trials and Research

Nedosiran is currently being studied in clinical trials to evaluate its safety and effectiveness. These trials involve different age groups and focus on various aspects of the drug’s performance[2]:

  • Pediatric Study: One ongoing study is looking at nedosiran in children from birth to 11 years old who have Primary Hyperoxaluria and relatively good kidney function.
  • Expanded Access Program: Another program is providing access to nedosiran for eligible patients with PH1 until the drug becomes commercially available or the program is terminated.

These studies aim to understand how well nedosiran works in reducing oxalate levels in the body and its overall impact on kidney function[2].

Safety and Monitoring

As with any investigational drug, the safety of nedosiran is being closely monitored. During clinical trials, researchers are tracking various safety measures[2]:

  • Adverse events (side effects)
  • Changes in vital signs (temperature, pulse rate, respiratory rate, blood pressure)
  • Changes in ECG (heart rhythm test) results
  • Changes in laboratory test results (blood tests, urine tests)
  • Physical examination findings

Patients participating in nedosiran trials are typically required to attend regular clinic visits for these safety assessments[1][2].

Potential Benefits

While the full benefits of nedosiran are still being studied, researchers are looking at several potential positive outcomes[2]:

  • Reduced oxalate levels: The main goal is to lower the amount of oxalate in urine and blood.
  • Improved kidney function: By reducing oxalate, nedosiran may help preserve kidney function.
  • Fewer kidney stones: Lower oxalate levels may lead to fewer kidney stone events.
  • Better quality of life: Researchers are assessing whether nedosiran can improve overall quality of life for patients with PH.

It’s important to note that as an investigational drug, the full range of benefits and potential risks of nedosiran are still being determined through ongoing research[1][2].

Aspect Details
Drug Name Nedosiran (also known as DCR-PHXC)
Target Condition Primary Hyperoxaluria (PH), particularly Type 1 (PH1)
Administration Monthly subcutaneous injection
Dosage Adults and adolescents ≥50 kg: 170 mg
Adults and adolescents <50 kg: 136 mg
Children 6-11 years: 3.5 mg/kg (max 170 mg)
Age Groups Studied Adults, adolescents, and children from birth to 11 years
Primary Outcomes Changes in urinary oxalate-to-creatinine ratio
Secondary Outcomes Safety profile, changes in plasma oxalate, kidney function (eGFR), stone events, quality of life
Study Duration Approximately 15 months

FAQ

  • What is Nedosiran? Nedosiran is an investigational drug being studied for the treatment of Primary Hyperoxaluria, particularly Type 1 (PH1). It is administered through subcutaneous injection (under the skin) once a month.
  • How is Nedosiran administered in clinical trials? In the trials, Nedosiran is given as a monthly subcutaneous injection. The dose varies based on the patient's age and weight. For adults and adolescents weighing 50 kg or more, the dose is 170 mg. For those weighing less than 50 kg, it's 136 mg. Children aged 6-11 receive 3.5 mg/kg, not exceeding 170 mg.
  • What are the main goals of the Nedosiran clinical trials? The main goals are to evaluate the safety, efficacy, and pharmacokinetics of Nedosiran in patients with Primary Hyperoxaluria. Researchers are particularly interested in its effects on urinary oxalate levels, kidney function, and overall quality of life for patients.
  • Are there any specific trials for children? Yes, there is a Phase 2 open-label study specifically for children from birth to 11 years of age with Primary Hyperoxaluria and relatively intact renal function. This study aims to assess the safety, efficacy, and appropriate dosing for younger patients.
  • What are some of the outcomes being measured in these trials? The trials are measuring various outcomes, including changes in urinary oxalate-to-creatinine ratio, plasma oxalate concentration, kidney stone formation, estimated glomerular filtration rate (eGFR), and quality of life measures. They are also closely monitoring safety through adverse events, physical exams, vital signs, and laboratory tests.

Ongoing Clinical Trials on Nedosiran

  • Study of nedosiran for patients with primary hyperoxaluria type 1 and severe kidney impairment with or without dialysis

    Recruiting

    1 1
    Investigated drugs:
    Germany Italy Spain

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

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Italy The Netherlands Norway Spain

Glossary

  • Primary Hyperoxaluria (PH): A rare genetic disorder characterized by overproduction of oxalate in the liver, leading to kidney and bladder stones, kidney damage, and other systemic problems.
  • Subcutaneous injection: A method of administering medication by injecting it into the layer of tissue between the skin and muscle, typically in the thigh or abdomen.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Oxalate: A compound found in many foods that, when overproduced or accumulated, can lead to kidney stones and other health issues.
  • eGFR (estimated Glomerular Filtration Rate): A test used to check how well the kidneys are working by estimating how much blood passes through the glomeruli (tiny filters in the kidneys) each minute.
  • Urinary oxalate-to-creatinine ratio: A measurement used to assess the amount of oxalate in urine relative to creatinine, which helps in diagnosing and monitoring conditions like Primary Hyperoxaluria.
  • Open-label study: A type of clinical trial where both the researchers and participants know which treatment is being administered.
  • Adverse event: Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.

References

  1. https://clinicaltrials.gov/study/NCT05993416
  2. https://clinicaltrials.gov/study/NCT05001269