Pelizaeus-Merzbacher disease – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:14:04 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Pelizaeus-Merzbacher disease – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Studying the Effects of Deferiprone on Motor Function in Children with Pelizaeus-Merzbacher Disease https://clinicaltrials.eu/trial/studying-the-effects-of-deferiprone-on-motor-function-in-children-with-pelizaeus-merzbacher-disease/ Wed, 29 Apr 2026 15:02:38 +0000 https://clinicaltrials.eu/trial/studying-the-effects-of-deferiprone-on-motor-function-in-children-with-pelizaeus-merzbacher-disease/ This clinical trial is focused on studying the effects of a medication called deferiprone in children with a rare genetic disorder known as Pelizaeus-Merzbacher disease (PMD). PMD affects the nervous system and can lead to problems with movement and coordination. The medication being tested, deferiprone, is taken as an oral solution, which means it is a liquid that is swallowed.

The purpose of this study is to explore how deferiprone might help improve the ability to move in children with PMD. During the study, participants will take the medication and their progress will be monitored over time. This will involve regular check-ups and assessments to see how their motor skills, such as sitting and moving, are affected by the treatment. Some participants may receive a placebo, which looks like the medication but does not contain the active ingredient.

The study will also include various tests to gather more information about the effects of deferiprone. These tests may involve brain scans, such as MRI, and other assessments to evaluate general health, quality of life, and specific abilities like hand function and communication. The goal is to understand whether deferiprone can make a positive difference for children with PMD and to gather data that could help in developing future treatments.

]]> Study on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ion356-for-patients-with-pelizaeus-merzbacher-disease/ Wed, 29 Apr 2026 14:26:19 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ion356-for-patients-with-pelizaeus-merzbacher-disease/ This clinical trial is focused on studying a rare genetic disorder called Pelizaeus-Merzbacher Disease (PMD). PMD affects the nervous system and is caused by a problem with the PLP1 gene. The study will use a treatment called ION356, which is a special type of medicine known as an antisense oligonucleotide. This medicine is designed to target and work on the genetic level to help manage the disease. ION356 is given as an injection directly into the space around the spinal cord, a method known as intrathecal use.

The main goal of this study is to evaluate the safety and tolerability of ION356 in patients with PMD. Participants will receive the treatment and be monitored for any side effects or changes in their health. The study will also look at how the body processes the medicine, including how it moves through the blood and spinal fluid, and how it is eliminated from the body. This will help researchers understand the best way to use ION356 for treating PMD.

Throughout the study, participants will have regular check-ups, which may include blood tests, urine tests, and other health assessments to ensure their safety. The study is expected to continue for several years, allowing researchers to gather important information about the long-term effects and benefits of ION356 for individuals with Pelizaeus-Merzbacher Disease.

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