RecruitingOBSERVATIONAL

Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.

This study focuses on people in France who have a rare genetic kidney condition called Primary Hyperoxaluria Type 1 (PH1). PH1 causes a build-up of a substance called oxalate, leading to kidney stones and kidney damage. This study is tracking patients who are taking a specific medicine called Lumasiran. Doctors want to understand how Lumasiran works for patients over a longer period, especially since previous studies were shorter. They will follow both children and adults for five years, checking their health, blood tests, and kidney scans regularly. The main goal is to see how the amount of oxalate in their body changes before and after they start Lumasiran treatment, and to get a clearer picture of the medicine's long-term effects and how best to manage patient care.

At a glance

Status

Recruiting

Sponsor

Hospices Civils de Lyon

Enrolment target

100

Start

01 Jan 2023

Estimated completion

31 Dec 2026

Patients With PH1 Treated With Lumasiran in France

What is this study about?

This study is all about a rare genetic condition called Primary Hyperoxaluria Type 1 (PH1). If you have PH1, your body makes too much of a substance called oxalate, which can build up and cause painful kidney stones, damage to your kidneys, and even kidney failure.

Doctors are interested in a medicine called Lumasiran, which has been shown to help treat PH1. While we know Lumasiran can be effective, earlier studies didn't follow patients for a very long time. This new study in France will track people receiving Lumasiran over five years – both children and adults. The goal is to get a much better idea of how well the medicine works in the long run, if there are any side effects over time, and how doctors can best care for patients taking it.

Essentially, the study wants to see how the levels of oxalate in a patient's body change before they start Lumasiran and after they've been on it for a while. By carefully monitoring patients' health, blood tests, and kidney scans over these five years, researchers hope to learn important information about managing PH1 with Lumasiran and improving the lives of those living with this condition.

Key takeaways

The study tracks people with a rare kidney condition (PH1) who are taking Lumasiran.
It aims to understand Lumasiran's long-term effects on kidney health over five years.
Data will be collected from routine medical check-ups, blood tests, and scans.
Both children and adults in France are included and will be followed.
The goal is to improve care and understanding for all PH1 patients.

Who may be eligible?

This study is looking for people who have Primary Hyperoxaluria Type 1 (PH1) and are currently being treated with Lumasiran in France. This includes patients who started Lumasiran when it was first available on a special access programme (called ATU) and those who started it after it became more widely available.

There are only a few reasons why someone wouldn't be able to join. If you, or your parents/guardians if you are under 18, don't want to take part in the study, then you wouldn't be included. Also, since this study is happening in France and tracking long-term health, you need to be covered by the French social security system (their national health insurance).

If you meet these requirements – you have PH1, are taking Lumasiran in France, and are covered by social security – then you might be able to participate. The study is open to people of all ages, from babies to adults up to 99 years old, and includes both males and females.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have Primary Hyperoxaluria Type 1 (PH1)?
Am I currently being treated with Lumasiran?
Do I live in France and am I covered by French social security?
Am I (or my legal guardian) willing to allow my medical information to be used for this study?

Answer every question to see your result.

What does participation involve?

If you join this study, it means that doctors will regularly check on your health and your PH1 condition over a five-year period. You won't be given new medication as part of this study; it's about observing how Lumasiran, which you're already taking, works over time. This means doctors will collect information from your usual visits, including blood tests and kidney scans (like ultrasounds), to monitor your health and how your oxalate levels are changing.

These checks will happen at regular intervals as part of your standard medical care, so it shouldn't add many extra appointments to your schedule. The study will track your progress over a total of five years, using information gathered from both your past medical records and your ongoing doctor visits. The aim is to get a full picture of your health journey with PH1 and Lumasiran.

Potential risks and benefits

The potential benefits of taking part in this study are mainly for the wider PH1 community, as the information gathered will help doctors better understand the long-term effects of Lumasiran and how to care for patients with PH1. For you as an individual, it provides careful, standardised monitoring of your condition, though you won't receive new treatments or changes to your care that aren't already part of your usual medical plan. Since you're already taking Lumasiran, there are no new medication risks associated with joining the study itself. Doctors will be collecting information from your existing medical visits, so the risks are generally minimal, not adding extra tests beyond your standard care. Remember, you can choose to withdraw from the study at any time without affecting your medical treatment.

Locations (7)

CHU de Besançon
Verified postcode
Besançon, France· Recruiting
Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant
Verified postcode
Bron, France· Recruiting
Hopital Edouard Herriot
Verified postcode
Lyon, France· Recruiting
AP-HM - Timone Enfants
Verified postcode
Marseille, France· Recruiting
Hôpital Européen G. Pompidou
Verified postcode
Paris, France· Recruiting
CHU Paris - Hôpital Necker-Enfants Malades
Verified postcode
Paris, France· Recruiting
Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres
Verified postcode
Paris, France· Recruiting

Common questions

What is Primary Hyperoxaluria Type 1 (PH1)?

It's a rare genetic condition where your body produces too much oxalate, leading to kidney stones and kidney damage.

What is Lumasiran?

Lumasiran is a medicine used to treat Primary Hyperoxaluria Type 1 (PH1) by helping reduce the amount of oxalate your body makes.

Will I get a new medicine if I join this study?

No, this study is for people already taking Lumasiran. You won't be given any new medicines as part of your participation.

How long will the study follow me?

The study will follow patients for five years to see the long-term effects of Lumasiran.

Does this study involve extra hospital visits?

Not usually. Information will be collected from your existing, regular doctor's appointments and check-ups.

How to find out more

Mélissa CLOAREC, Clinical Research Associate

melissa.cloarec@chu-lyon.fr 04 27 85 51 54 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.