Ataxia telangiectasia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Sat, 20 Jun 2026 04:04:01 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Ataxia telangiectasia – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of N-Acetyl-L-Leucine in Patients with Ataxia-Telangiectasia: A Phase III Trial https://clinicaltrials.eu/trial/study-on-the-effects-of-n-acetyl-l-leucine-for-patients-with-ataxia-telangiectasia/ Wed, 29 Apr 2026 15:04:08 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-n-acetyl-l-leucine-for-patients-with-ataxia-telangiectasia/ This study focuses on testing a medication called N-Acetyl-L-Leucine in patients with Ataxia-Telangiectasia (A-T), a rare genetic condition that affects body movements and coordination. The medication is given as an oral suspension, which means it is a liquid medicine taken by mouth.

The purpose of this research is to determine how well N-Acetyl-L-Leucine works in treating the movement problems associated with Ataxia-Telangiectasia. During the study, participants will receive either the study medication or a placebo at different times. The treatment period lasts for 12 months, and the maximum daily dose of the medication is 4 grams.

This is a special type of study called a crossover study, which means participants will switch between receiving the actual medication and placebo during different periods of the study. Neither the patients nor the doctors will know when participants are receiving the actual medication or placebo, as this helps ensure accurate results.

]]> Study on the Effects of EryDex (Dexamethasone Sodium Phosphate) for Children Aged 6-9 with Ataxia Telangiectasia https://clinicaltrials.eu/trial/study-on-the-effects-of-erydex-dexamethasone-sodium-phosphate-for-children-aged-6-9-with-ataxia-telangiectasia/ Wed, 29 Apr 2026 14:29:51 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-erydex-dexamethasone-sodium-phosphate-for-children-aged-6-9-with-ataxia-telangiectasia/ This clinical trial is focused on studying the effects of a treatment called EryDex on individuals with Ataxia Telangiectasia, a rare genetic disorder that affects the nervous system and other parts of the body. The treatment involves using a medication called Dexamethasone sodium phosphate, which is encapsulated into the patient’s own red blood cells. This process is designed to help manage symptoms related to the central nervous system, such as coordination and movement difficulties.

The purpose of the study is to evaluate how effective EryDex is in improving neurological symptoms in children aged 6 to 9 years with Ataxia Telangiectasia. Participants in the study will be randomly assigned to receive either the EryDex treatment or a placebo. The study is conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure the results are unbiased and reliable.

Throughout the study, participants will undergo regular assessments to monitor changes in their symptoms. The study will last for several months, with visits scheduled to track progress and gather data on the treatment’s effects. The ultimate goal is to determine whether EryDex can provide meaningful improvements in the quality of life for those living with Ataxia Telangiectasia.

]]> Study on the Safety and Effects of EryDex with Dexamethasone Sodium Phosphate for Patients with Ataxia Telangiectasia https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-erydex-with-dexamethasone-sodium-phosphate-for-patients-with-ataxia-telangiectasia/ Wed, 29 Apr 2026 14:22:45 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-erydex-with-dexamethasone-sodium-phosphate-for-patients-with-ataxia-telangiectasia/ This clinical trial is focused on studying a rare genetic disorder called Ataxia Telangiectasia (A-T). A-T affects the nervous system and other parts of the body, leading to problems with movement and coordination. The study will use a treatment called EryDex, which involves the use of a medication named Dexamethasone sodium phosphate. This medication is encapsulated into the patient’s own red blood cells and then infused back into the body. The purpose of the study is to evaluate the safety and tolerability of EryDex in patients with A-T and to explore its effects on neurological symptoms.

Participants in this study will have previously completed another study related to A-T. During the course of the study, participants will receive the EryDex treatment and be monitored for any side effects or changes in their health. The study will assess any treatment-emergent adverse events, which are any new or worsening medical issues that occur during the study. This includes serious adverse events, changes in vital signs, and findings from physical and neurological examinations.

The study aims to provide valuable information on how EryDex affects patients with A-T, particularly in terms of safety and its potential impact on neurological symptoms. The study will continue until 2026, allowing researchers to gather comprehensive data on the treatment’s effects over time.

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