Angelman’s syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 24 Jun 2026 04:16:03 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Angelman’s syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A Phase 3 study to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome https://clinicaltrials.eu/trial/a-phase-3-study-to-evaluate-the-efficacy-and-safety-of-ion582-in-children-and-adults-with-angelman-syndrome/ Wed, 29 Apr 2026 15:09:22 +0000 https://clinicaltrials.eu/trial/a-phase-3-study-to-evaluate-the-efficacy-and-safety-of-ion582-in-children-and-adults-with-angelman-syndrome/ This study aims to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome. This condition is a rare genetic disorder typically caused by a deletion, which is a missing piece of genetic material, or a mutation, which is a change in the DNA sequence, of the UBE3A gene. The study focuses on how this specific genetic change affects the body and brain.

The treatment being investigated is ION582, which is a type of antisense oligonucleotide. This is a specialized substance designed to target and interact with specific parts of genetic instructions to help manage the effects of the disease. The medication will be delivered through an intrathecal injection, which is a method where the medicine is injected into the fluid surrounding the brain and spinal cord.

During the study, participants will receive the medication and undergo regular follow-up periods to monitor how they are doing. The duration of the observation for participants is approximately 52 weeks. This process allows for the monitoring of various aspects of health and development over time.

]]> A Study Testing the Safety and How Well GTX-102 Works in Adults and Children with Angelman Syndrome https://clinicaltrials.eu/trial/a-study-testing-the-safety-and-how-well-gtx-102-works-in-adults-and-children-with-angelman-syndrome/ Wed, 29 Apr 2026 15:08:44 +0000 https://clinicaltrials.eu/trial/a-study-testing-the-safety-and-how-well-gtx-102-works-in-adults-and-children-with-angelman-syndrome/ This study is looking at a condition called Angelman Syndrome, which is a genetic disorder that affects the nervous system and causes problems with development, movement, balance, speech, and sleep. The study will test a treatment called GTX-102, also known as apazunersen, which is a type of medicine designed to work at the genetic level to address the underlying cause of the condition. The medicine will be given along with a solution called GTX/UX Diluent and Flush Solution, which helps to deliver and clear the treatment. Both the treatment and the solution will be given through a procedure called intrathecal use, which means the medicine is injected into the space around the spinal cord. The study includes people with different types of Angelman Syndrome, including deletion-type, nondeletion-type, and mutation-type forms of the condition.

The purpose of this study is to look at how safe GTX-102 is and whether it helps improve symptoms in people with Angelman Syndrome. The study will examine different age groups and types of the condition separately. For younger children with deletion-type Angelman Syndrome, the study will measure thinking skills. For older children with certain types of the condition, the study will look at multiple areas including thinking, communication, behavior, sleep, and movement. For adults with any type of Angelman Syndrome, the study will focus on communication skills, behavior, and movement abilities. The study will track any side effects that occur and how severe they are.

People taking part in the study will receive the treatment for up to 48 weeks. During this time, they will have regular visits where doctors will check their progress using various tests and assessments. These assessments will measure changes in cognitive abilities, communication skills, motor function, behavior, and sleep patterns. The study will use several standard tools to measure these changes, and will compare how people are doing at different points during the treatment compared to when they started. The study is expected to continue until 2030 and will include both children and adults with Angelman Syndrome.

]]> Study on the Effects of GTX-102 for Children with Angelman Syndrome https://clinicaltrials.eu/trial/study-on-the-effects-of-gtx-102-for-children-with-angelman-syndrome/ Wed, 29 Apr 2026 15:03:33 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-gtx-102-for-children-with-angelman-syndrome/ This clinical trial is focused on studying Angelman Syndrome, a rare genetic disorder that affects the nervous system and causes developmental disabilities and neurological problems. The study will investigate the effects of a treatment called GTX-102, which is a solution for injection designed to improve cognitive function in children with Angelman Syndrome. The treatment involves a special type of medication known as an antisense oligonucleotide, which is a small piece of DNA or RNA that can bind to specific molecules in the body to alter their function.

The purpose of the study is to evaluate how well GTX-102 works in improving cognitive abilities in children with Angelman Syndrome. Participants in the study will receive the treatment through an injection into the space around the spinal cord, a method known as intrathecal use. The study will also use a solution called GTX/UX Diluent and Flush Solution to help administer the treatment. This solution contains several common substances, including sodium chloride and potassium chloride, which are often used in medical settings to maintain the balance of fluids and electrolytes in the body.

Throughout the study, participants will be monitored for changes in their cognitive abilities and any potential side effects. The study will last for a period of time, during which participants will have regular visits to assess their progress. The goal is to determine if GTX-102 can provide a meaningful improvement in the lives of children with Angelman Syndrome by enhancing their cognitive function and overall quality of life.

]]> Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome https://clinicaltrials.eu/trial/study-on-the-long-term-safety-of-gtx-102-for-patients-with-angelman-syndrome/ Wed, 29 Apr 2026 15:01:42 +0000 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-of-gtx-102-for-patients-with-angelman-syndrome/ This clinical trial is focused on studying the long-term safety and effectiveness of a treatment called GTX-102 for individuals with Angelman Syndrome. Angelman Syndrome is a genetic disorder that affects the nervous system and can cause developmental delays, speech difficulties, and balance issues. The treatment being tested, GTX-102, is a special type of medication known as an antisense oligonucleotide, which is designed to target specific genetic material in the body. It is administered as a solution for injection directly into the spinal fluid, a method known as intrathecal use.

The purpose of this study is to evaluate how safe GTX-102 is for patients over a long period. Participants in the trial will receive the treatment and be monitored for any side effects or changes in their condition. The study will also involve the use of Elliotts B Solution, a buffered intrathecal electrolyte/dextrose injection, which contains substances like calcium chloride, glucose, and sodium chloride. This solution is used to help deliver the treatment safely.

Throughout the study, participants will undergo regular assessments to track their progress and any changes in their symptoms. These assessments will include evaluations of cognitive abilities, communication skills, and motor functions. The study aims to provide valuable information on the long-term use of GTX-102 in managing Angelman Syndrome, helping to determine its potential benefits and any risks associated with its use.

]]> Study on the Safety and Effects of ION582 for Patients with Angelman Syndrome https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ion582-for-patients-with-angelman-syndrome/ Wed, 29 Apr 2026 15:00:06 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ion582-for-patients-with-angelman-syndrome/ This clinical trial is focused on studying Angelman syndrome, a rare genetic disorder that affects the nervous system and causes developmental disabilities and neurological problems. The study will use a treatment called ION582, which is an injection designed to target specific genetic material in the body. The treatment involves a type of medication known as an antisense oligonucleotide, which is a small piece of DNA or RNA that can bind to specific molecules in the body to alter their function.

The purpose of the study is to evaluate the safety and tolerability of ION582 when administered through injections into the spinal fluid, a method known as intrathecal use. Participants will receive multiple doses of the medication, and researchers will monitor them for any side effects or changes in their health. The study will also look at how the body processes the medication, including how long it stays in the bloodstream and how it is eliminated from the body.

Throughout the study, participants will undergo regular health checks, including monitoring of vital signs and laboratory tests, to ensure their safety. The study aims to gather important information about the potential benefits and risks of ION582 for individuals with Angelman syndrome, which could lead to new treatment options in the future.

]]> Study on the Safety and Tolerability of Rugonersen in Patients with Angelman Syndrome https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-rugonersen-in-patients-with-angelman-syndrome/ Wed, 29 Apr 2026 14:23:14 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-rugonersen-in-patients-with-angelman-syndrome/ This clinical trial is focused on studying Angelman Syndrome, a rare genetic disorder that affects the nervous system and causes developmental disabilities and neurological problems. The study will use a treatment called Rugonersen, which is administered as a solution for injection. The purpose of the study is to investigate the safety and tolerability of Rugonersen in individuals with Angelman Syndrome.

Participants in the study will receive the treatment through a method called intratracheal use, which involves delivering the medication directly into the windpipe. The study will monitor participants over a period of time to observe any side effects or changes in their health. This will help researchers understand how the body processes the medication and how it affects the participants’ health and well-being.

The study will also collect information on the frequency and severity of any adverse events, such as unexpected health issues, and will track changes in vital signs like blood pressure and heart rate. By gathering this data, researchers aim to ensure the treatment is safe and to learn more about its potential benefits for people with Angelman Syndrome.

]]> ASA https://clinicaltrials.eu/organisation/asa/ Wed, 29 Apr 2026 13:34:16 +0000 https://clinicaltrials.eu/organisation/asa/ The Asociación Síndrome de Angelman is a non-profit organization, declared to be of public utility in 2014. Founded in Barcelona in October 1996 by parents of children with Angelman Syndrome, its core mission is to support families by fostering communication, offering advice, and providing essential information. The association actively promotes research into Angelman Syndrome at all levels to deepen understanding and is dedicated to enhancing the quality of life for individuals affected by the condition. While founded in Barcelona, the association’s office is located in Madrid, and it serves families from across Spain.

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