Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Long-Term follow-up Study to evaluate safety and tolerability of olipudase alfa in patients who completed the DFI12712 or the LTS13632 Study in France - PTA17397

This study, called PTA17397, is a long-term follow-up to check on the safety of a medicine called Xenpozyme (also known as olipudase alfa). It's designed for people with Niemann-Pick disease who have previously taken part in two other studies, DFI12712 or LTS13632. The main goal is to understand how well people tolerate Xenpozyme over an extended period. Researchers will be carefully monitoring for any unwanted effects, often called 'adverse events,' to make sure the treatment continues to be safe for patients. This type of study, often called a 'Phase II' study, helps doctors learn more about promising new treatments.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Sanofi Winthrop Industrie

Enrolment target

Start

02 Aug 2024

Niemann-Pick disease

What is this study about?

This research study is called a 'long-term follow-up' study. This means it's designed to keep an eye on people for a long time after they've already taken part in other studies about a medicine. In this case, the medicine is called Xenpozyme (also known by its scientific name, olipudase alfa). This medicine is being developed to help people with Niemann-Pick disease.

The main purpose of this study is to see how safe Xenpozyme is over many years. Doctors want to know if people continue to tolerate the medicine well and if there are any long-term side effects or problems. They will be counting any 'adverse events' – which are any unexpected or unwanted health problems that happen during the study, regardless of whether they are related to the medicine.

This study is important because it helps ensure that treatments for Niemann-Pick disease are not only effective but also safe for people to take over a lifetime. By following people who have already been treated with Xenpozyme, doctors can gather vital information about its long-term effects and help make sure it's the best and safest option for patients.

Key takeaways

This study is about the long-term safety of Xenpozyme for Niemann-Pick disease.
It's for people who have already taken part in two specific previous studies (DFI12712 or LTS13632).
Doctors will closely monitor for any side effects or health problems over time.
It aims to build a full picture of the medicine's safety profiles over many years.
Participation requires you to be at least 18 years old, with no upper age limit.

Who may be eligible?

To be able to join this study, you must have Niemann-Pick disease and have already taken part in one of two specific previous studies: either study DFI12712 or study LTS13632. These previous studies would have involved you receiving the medicine Xenpozyme.

There is no upper age limit for taking part, but you must be at least 18 years old. Both men and women are welcome to participate. The study is particularly looking for people in France.

If you meet these requirements, and your doctor thinks it's a good idea for you to continue being monitored as part of this long-term assessment, then you might be able to take part.

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.

I have Niemann-Pick disease.
I am at least 18 years old.
I have previously taken part in the DFI12712 study.
OR I have previously taken part in the LTS13632 study.
I am currently in France.

Answer every question to see your result.

What does participation involve?

Taking part in this study means you would remain on the medicine Xenpozyme. You would have regular check-ups with your study doctor and nurses. During these visits, the medical team would carefully monitor your health to see how you are tolerating the medicine.

This would involve discussions about any health problems or side effects you might be experiencing. They would also likely conduct physical examinations and possibly some tests, like blood tests, to keep track of your health. The duration of your participation in this long-term follow-up study would depend on the study plan, but it's designed to track safety over an extended period.

Potential risks and benefits

A potential benefit of joining this study is that you would continue to receive Xenpozyme, which is a treatment for Niemann-Pick disease, and your health would be closely monitored by a medical team experienced in Niemann-Pick disease. This close monitoring could help detect and manage any health issues quickly. As with any medicine, there are potential risks, including the possibility of experiencing side effects from Xenpozyme. These will be carefully explained to you by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

—
Unverified
France

Common questions

What is Niemann-Pick disease?

Niemann-Pick disease is a rare inherited condition that affects the body's ability to break down and remove certain fatty substances, leading to their build-up in organs like the brain, spleen, and liver.

What is Xenpozyme?

Xenpozyme (also called olipudase alfa) is a medicine being studied as a treatment for Niemann-Pick disease. It's designed to help your body break down the fatty substances that build up in the disease.

What does 'long-term follow-up' mean?

It means the study will check on your health and how you react to the medicine over many years, even after the main part of the original study finished.

Why is this study only for people already in other studies?

This study is a continuation: it's designed to gather more safety information from people who have already been taking Xenpozyme in earlier research studies.

What are 'adverse events'?

Adverse events are any unwanted medical problems that happen during a study, whether or not they are thought to be related to the medicine being tested.