Not recruitingRare disease

A Study to Evaluate the Safety of Olipudase Alfa in Children and Adults with Niemann-Pick Disease Who Completed Previous Treatment Studies

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What is this trial about?

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

This study involves patients with Niemann-Pick disease, a rare inherited condition where a specific substance builds up in various organs of the body because the body lacks an enzyme needed to break it down. The treatment being studied is Xenpozyme, also known by its active ingredient name olipudase alfa or code name GZ402665, which is given as an infusion into a vein. This medication is designed to replace the missing enzyme in patients with acid sphingomyelinase deficiency, which is the underlying cause of this form of Niemann-Pick disease.

The purpose of this study is to assess safety and tolerability of the treatment in patients who have already completed previous studies with this medication in France. The study will continue until the medication receives reimbursement approval in France or for a maximum of five years, whichever happens first. Patients will receive the medication through intravenous use, meaning it will be delivered directly into the bloodstream through a vein.

During the study, participants will continue receiving regular infusions of the medication, and doctors will monitor them for any unwanted effects or health changes. The study is following patients who were already receiving this treatment in earlier research studies to gather more information about how safe the medication is when used over a longer period of time. The medication comes as a powder for concentrate for solution for infusion, which means it needs to be mixed with liquid before being given to the patient.

The research process

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

Step 1
Enrollment and initial assessment
After completing the previous study (DFI12712 or LTS13632), your participation in this long-term follow-up study will begin.
You will be asked to sign an informed consent form, which is a document confirming that you agree to participate in the study and understand what it involves.
If you are a female of childbearing potential, a pregnancy test (urine test) will be performed to confirm that you are not pregnant before starting the study.
You will need to confirm that you are willing to follow the study procedures and requirements.
Step 2
Treatment period with olipudase alfa
You will receive olipudase alfa (also known as Xenpozyme), which is the medication being evaluated in this study.
The medication will be given to you through an intravenous infusion, which means it will be administered directly into your vein through a needle.
The medication comes as a powder that will be mixed into a solution before being given to you.
The treatment will continue until olipudase alfa is approved for reimbursement in France or for a maximum of 5 years, whichever occurs first.
The specific dosage and frequency of infusions will be determined based on your individual treatment plan from the previous study.
Step 3
Safety monitoring throughout the study
During the entire treatment period, your health will be monitored to assess the safety and tolerability of the medication.
Any adverse events (unwanted or unexpected symptoms or health problems) will be recorded and evaluated.
Any serious adverse events (health problems that are life-threatening, require hospitalization, or cause significant disability) will also be carefully monitored and documented.
You should report any new symptoms or health changes to the study staff during your visits.
Step 4
Contraception requirements (if applicable)
If you are a sexually active female of childbearing potential or a sexually active male, you will need to use contraception (birth control methods) during the study.
You must either practice true abstinence (not having sexual intercourse) in line with your usual lifestyle, or use 2 effective methods of contraception.
This requirement applies for the entire treatment period and for at least 28 days after receiving the last dose of the study medication.
Step 5
Study completion
The study will end when olipudase alfa receives reimbursement approval in France or after 5 years of treatment, whichever comes first.
Your final assessment will be completed at the end of the study period.

Who can join the trial?

5 criteria

The patient must have finished participating in a previous study called DFI12712 or LTS13632 in France
The patient must sign a consent form, which is a document showing they agree to take part in the study, before any study activities begin
The patient must be willing to follow all the rules and requirements of the study
Female patients who are able to become pregnant must have a negative pregnancy test, which is a urine test that checks if someone is pregnant, when joining the study
Female patients who are able to become pregnant and are sexually active, as well as male patients who are sexually active, must either not have sexual intercourse or use two reliable methods of birth control, which are ways to prevent pregnancy, during the entire treatment period and for at least 28 days after receiving the last dose of the study medicine

Who cannot join the trial?

4 criteria

The study has not provided specific exclusion criteria information in the available data
You should discuss with your doctor whether you meet the requirements to participate in this study
This study is specifically designed for patients who have already completed previous studies called DFI12712 or LTS13632
If you have not participated in either of these earlier studies, you may not be eligible for this study

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Groupe Hospitalier Diaconesses Croix Saint Simon
Paris, France

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Investigated drugs

Olipudase alfa is a medication being tested for the treatment of ASMD, which is a rare genetic disease. This medicine works by replacing an enzyme that is missing or not working properly in people with this condition. The enzyme helps break down certain fats that build up in the body's organs and tissues. This treatment is given to help reduce these harmful fat deposits and improve symptoms in patients with ASMD.

What is already known about the treatment

Olipudase alfa – Olipudase alfa is an enzyme replacement therapy administered through intravenous infusion directly into the bloodstream, currently under investigation in long-term clinical studies for patients with a rare genetic condition called Niemann-Pick disease. This medication works by replacing a missing or deficient enzyme called acid sphingomyelinase, which normally breaks down a fatty substance called sphingomyelin in the body's cells; when this enzyme is lacking, sphingomyelin builds up in organs and causes damage. The study aims to evaluate the long-term safety and tolerability of this treatment in patients who have already participated in previous clinical trials. Olipudase alfa belongs to the class of enzyme replacement therapies designed to treat specific metabolic disorders caused by enzyme deficiencies.

Investigated diseases

Niemann-Pick Disease – Niemann-Pick disease is a rare inherited disorder that affects the body's ability to break down and use fats and cholesterol within cells. The condition occurs when a specific enzyme needed to process lipids is missing or does not work properly. As a result, harmful amounts of fatty substances accumulate in various organs including the liver, spleen, lungs, and brain. The disease typically begins in childhood, though some forms may appear later in life. Symptoms often include an enlarged liver and spleen, difficulty with coordination and movement, and respiratory problems. The progression and severity of the disease varies depending on the specific type and can affect multiple body systems over time.