RecruitingPHASE1INTERVENTIONAL

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

The 'Orbit Study' is testing a new medicine called ION356 for a rare condition called Pelizaeus-Merzbacher Disease (PMD). This condition affects boys, usually between 2 and 17 years old, because of a specific genetic problem. The main goal of this early-stage study is to check how safe ION356 is and if the boys taking it experience any side effects. Researchers also want to understand how the body uses and processes the medicine. About 24 boys with PMD who have a specific gene change (PLP1 duplication) will take part. The study will last for a total of nearly three years, starting with a main testing period and then moving into a longer follow-up period.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Ionis Pharmaceuticals, Inc.

Enrolment target

Start

10 Apr 2024

Estimated completion

01 Jun 2028

Pelizaeus-Merzbacher Disease

What is this study about?

The 'Orbit Study' is an important step in finding new ways to help boys with Pelizaeus-Merzbacher Disease (PMD). PMD is a rare, complex condition that affects the brain and spinal cord. It's caused by a problem with a specific gene, and this study is focusing on boys who have a double copy of this gene, called PLP1 duplication.

The main aim of this study is to test a new medicine called ION356. Think of it like taking the very first steps with a new medicine – researchers really want to understand if it's safe for children and how their bodies react to different amounts of it. This is called a 'Phase 1' study, meaning it's an early but crucial stage in developing any new treatment.

About 24 boys with PMD will take part. They will be given increasing amounts of ION356 over time, with doctors carefully watching for any effects, good or bad. After this initial testing period, there's a longer follow-up part of the study, so doctors can continue to track how the boys are doing. This careful process helps ensure that any potential medicine is thoroughly checked before it can be used more widely.

Key takeaways

This study is testing a new medicine (ION356) for boys with Pelizaeus-Merzbacher Disease (PMD) who have a specific genetic change.
The main goal is to check how safe ION356 is and how the body handles it.
It involves boys aged 2-17 and requires genetic confirmation of the PLP1 duplication.
The medicine is given directly into the spinal fluid.
The study lasts for nearly three years, with regular visits and tests.
Participation helps future research, but potential benefits to your child are not guaranteed.

Who may be eligible?

This study is specifically looking for boys with Pelizaeus-Merzbacher Disease (PMD). They need to be between 2 and 17 years old at the time they consider joining. It's important that their PMD has been confirmed by a genetic test showing a specific gene change called PLP1 duplication, and that their doctors agree their brain scans and symptoms fit with a PMD diagnosis.

A parent or legal guardian must be able to give permission and be fully involved, attending all appointments and sharing information about their child's health. The family also needs to be able to travel to the study clinic and follow all the study's instructions.

There are some reasons why a boy might not be able to join. For example, if he has other significant health problems, has recently been part of another drug trial, or has had certain types of surgery. The study also cannot include boys who would have difficulty getting an MRI scan or a spinal tap (lumbar puncture), as these are important parts of the study.

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.

Is my child a boy between 2 and 17 years old?
Does my child have a confirmed diagnosis of PMD with the PLP1 gene duplication?
Can we travel to the study clinic for regular appointments?
Am I, as a parent or guardian, able to attend appointments and give feedback about my child?
Is my child generally well and does he not have other serious health problems?
Has my child not taken part in another drug study recently?

Answer every question to see your result.

What does participation involve?

If you decide to take part, your child will receive doses of the study medicine, ION356, directly into their spinal fluid (the fluid around the brain and spinal cord). This is done through a procedure called a lumbar puncture (also known as a spinal tap).

The study starts with a 'multiple-ascending dose' part, which lasts about 48 weeks (nearly a year). During this time, your child will receive increasing doses of ION356, and doctors will carefully monitor how he responds. After this, if eligible, your child can move into a 'long-term extension' part of the study, which lasts for another 109 weeks (about two years). During this extension, he will continue to receive ION356.

Throughout the study, there will be regular visits to the study clinic, and your child will have various tests, including physical examinations, blood tests, and MRI scans. You will also be asked to provide feedback on your child's symptoms and general well-being. The total duration of participation could be nearly three years.

Potential risks and benefits

Participating in any study has both possible benefits and potential risks. It's possible that your child might not experience any direct benefits from this treatment, as it's an early-stage study primarily focused on safety. However, by taking part, you would be helping researchers learn more about PMD and a potential new treatment, which could benefit others in the future. The main risks are linked to receiving the medicine and the procedures involved, such as the lumbar puncture, which can have side effects like headache. While the study medicine is carefully tested, there could also be unexpected side effects. You have the right to withdraw your child from the study at any time, for any reason, without it affecting his medical care.

Locations (7)

Ionis Investigative Site
Verified postcode
Atlanta, United States· Recruiting
Ionis Investigative Site
Verified postcode
Philadelphia, United States· Recruiting
Ionis Investigative Site
Verified postcode
Salt Lake City, United States· Recruiting
Ionis Investigative Site
Verified postcode
Le Kremlin-Bicêtre, France· Recruiting
Ionis Investigative Site
Verified postcode
Göttingen, Germany· Recruiting
Ionis Investigative Site
Verified postcode
Tokyo, Japan· Recruiting
Ionis Investigative Site
Verified postcode
Amsterdam, Netherlands· Recruiting

Common questions

What is Pelizaeus-Merzbacher Disease (PMD)?

PMD is a rare genetic condition that affects the brain and spinal cord, impacting movement, coordination, and development in boys.

What is ION356?

ION356 is the new experimental medicine being tested in this study to see if it can help boys with PMD caused by a specific gene change.

How will the medicine be given?

The medicine will be given directly into the fluid around the brain and spinal cord through a procedure called a lumbar puncture (spinal tap).

How long will the study last?

The study will last for nearly three years in total, with an initial testing phase and then a longer follow-up period.

Will my child get the medicine for free?

Yes, if your child qualifies and joins the study, the study medicine will be provided free of charge.

How to find out more

Ionis Pharmaceuticals, Inc.

ionispelizaeusmerzbacherstudy2@clinicaltrialmedia.com (844) 387-9520 Official trial record

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