Study on Lumasiran for Patients with Advanced Primary Hyperoxaluria Type 1

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What is this study about?

This clinical trial is focused on studying a rare genetic condition called Primary Hyperoxaluria Type 1 (PH1). PH1 is a disorder that causes the body to produce too much of a substance called oxalate, which can lead to kidney stones and other serious health problems. The trial is testing a new treatment called Lumasiran, also known by its code name ALN-GO1. Lumasiran is a solution for injection that is designed to reduce the levels of oxalate in the body.

The purpose of the study is to evaluate how effective and safe Lumasiran is for patients with advanced PH1. The study is divided into two groups: one group includes patients who are not on dialysis, and the other group includes patients who are on dialysis. Dialysis is a treatment that helps remove waste products from the blood when the kidneys are not working properly. The study will look at how Lumasiran affects the levels of oxalate in the blood over a period of several months.

Participants in the study will receive regular injections of Lumasiran and will have their blood oxalate levels monitored. The study will also assess other health aspects, such as kidney function and quality of life, to understand the overall impact of the treatment. The trial aims to provide valuable information about the potential benefits of Lumasiran for people living with PH1.

1 initial screening

Upon joining the study, the first step involves an initial screening process. This includes confirming the diagnosis of primary hyperoxaluria type 1 (PH1) through genetic analysis. Blood samples are collected to measure plasma oxalate levels, which must be at least 20 micromoles per liter.

If applicable, confirmation of a stable vitamin B6 regimen for at least 90 days prior to the study is required. For patients in cohort B, a stable hemodialysis regimen for at least four weeks before the screening is necessary.

2 consent and assent

Participants or their legal guardians must provide written informed consent. For minors, assent is also required according to local and national regulations.

3 baseline assessment

A baseline assessment is conducted to establish initial health parameters. This includes measuring plasma oxalate levels and other relevant health indicators.

4 treatment administration

The investigational drug, lumasiran, is administered as a subcutaneous injection. The dosage is 94.5 mg/0.5 mL, delivered as a solution for injection. The frequency and duration of administration are determined by the study protocol and medical team.

5 follow-up visits

Regular follow-up visits are scheduled to monitor the effects of the treatment. These visits include blood tests to measure changes in plasma oxalate levels and assessments of overall health and kidney function.

For cohort B, additional monitoring between dialysis sessions is conducted to evaluate changes in plasma oxalate levels.

6 end of study assessment

At the end of the study period, a final assessment is conducted. This includes measuring the percent change in plasma oxalate levels from baseline to month 6, as well as evaluating any changes in quality of life and other health parameters.

Who Can Join the Study?

Must have reached at least 37 weeks of pregnancy (full-term infant) at the time of giving consent.
Must have documentation or confirmation of Primary Hyperoxaluria Type 1 (PH1) through genetic analysis before starting the study.
Patients aged 18 years or older must have an estimated Glomerular Filtration Rate (eGFR) of 45 mL/min/1.73 m² or less. This is a measure of kidney function. For patients aged 12 months to under 18 years, a different formula is used. For those under 12 months, the level of serum creatinine (a waste product in the blood) must be high for their age.
The average of the three most recent blood tests measuring plasma oxalate (a substance in the blood) before the first day of the study must be 20 micromoles per liter (µmol/L) or higher. For patients on dialysis, these tests can include samples taken before dialysis sessions.
If taking vitamin B6 (pyridoxine) as a treatment, must have been on a stable dose for at least 90 days before giving consent and be willing to continue this stable dose until at least the 6-month visit. Adjustments for weight gain are allowed.
Must be willing and able to follow the study requirements and provide written informed consent. If under the legal age for consent, a legal guardian must provide consent, and the patient should agree to participate according to local laws.
For patients in Cohort B only: Must be on a stable hemodialysis (a treatment for kidney failure) schedule for at least 4 weeks before the study’s blood test and be willing to continue this schedule until the 6-month visit. Changes to the dialysis schedule are allowed only if medically necessary.

Who Cannot Join the Study?

Patients who do not have Primary Hyperoxaluria Type 1 (PH1) cannot participate. This is a specific genetic condition that affects the body’s ability to process a substance called oxalate.
Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
Patients who are not willing or able to follow the study procedures cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate.
Patients who are currently participating in another clinical trial cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have a history of allergic reactions to the study medication cannot participate.
Patients who are unable to provide informed consent cannot participate. This means they must understand the study and agree to take part.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Hospices Civils De Lyon	Lyon	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Awvtpojzb Uqu	Amsterdam	The Netherlands
Cgslexsbb Usrrkggybunmaq Suojinrro	Woluwe-Saint-Lambert	Belgium

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	22.01.2020
France	Not recruiting	22.01.2020
Italy	Not recruiting	22.01.2020
The Netherlands	Not recruiting	22.01.2020

Trial locations

Investigated drugs:

Lumasiran

Lumasiran is a medication being studied for its ability to lower the levels of a substance called oxalate in the blood. Oxalate is a natural chemical in the body, but too much of it can cause health problems, especially in people with a condition called Primary Hyperoxaluria Type 1 (PH1). This condition can lead to kidney stones and other serious kidney issues. Lumasiran works by reducing the production of oxalate in the liver, which may help prevent these complications. The study is looking at how well Lumasiran works in patients with PH1, both those who are on dialysis and those who are not.

Investigated diseases:

Hyperoxaluria

Primary Hyperoxaluria Type 1 (PH1) – Primary Hyperoxaluria Type 1 is a genetic disorder that leads to the overproduction of oxalate, a substance that can form crystals and stones in the kidneys and urinary tract. This condition is caused by a deficiency of the liver enzyme alanine-glyoxylate aminotransferase (AGT), which normally helps to break down oxalate. As the disease progresses, the accumulation of oxalate can lead to kidney damage and the formation of kidney stones. Over time, if the kidneys are unable to filter out the excess oxalate, it can lead to systemic oxalosis, where oxalate deposits in other organs and tissues. This can affect the bones, eyes, heart, and skin, causing a range of symptoms. The severity and progression of the disease can vary widely among individuals.

Trial ID:

2023-503382-29-00

Protocol code:

ALN-GO1-005

NCT ID:

NCT04152200

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Lumasiran for Patients with Advanced Primary Hyperoxaluria Type 1

What is this study about?

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