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Study on the Safety and Effects of ION582 for Patients with Angelman Syndrome

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What is this trial about?

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

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.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm the diagnosis of Angelman syndrome. This involves reviewing medical history and ensuring the participant meets the inclusion criteria, such as age and current treatment stability.

  2. Step 2

    Baseline evaluation

    A baseline evaluation is performed to gather initial health data. This includes measuring vital signs and conducting laboratory tests to establish a reference point for future assessments.

  3. Step 3

    First dose administration

    The first dose of ION582 is administered via intrathecal injection. This means the medication is injected into the space around the spinal cord. The dosage is determined based on the study protocol.

  4. Step 4

    Monitoring and follow-up

    After the first dose, monitoring is conducted to observe any immediate effects. This includes checking for adverse reactions and changes in vital signs. Follow-up visits are scheduled to continue monitoring the participant's response to the medication.

  5. Step 5

    Subsequent dose administrations

    Additional doses of ION582 are administered at specified intervals. The frequency and dosage may vary as the study progresses, following the protocol's guidelines for ascending dose levels.

  6. Step 6

    Ongoing assessments

    Throughout the trial, regular assessments are conducted to evaluate the safety and tolerability of the medication. This includes monitoring for any adverse events and conducting laboratory tests to track changes in health parameters.

  7. Step 7

    Final evaluation

    At the end of the trial, a final evaluation is performed. This includes a comprehensive review of the participant's health status and any changes observed during the study. The data collected will contribute to understanding the effects of ION582 in treating Angelman syndrome.

Who can join the trial?

5 criteria

  • The participant must have a documented and certified diagnosis of Angelman syndrome. This means they have a specific genetic condition involving a deletion or mutation in a gene called UBE3A.
  • The participant can be a male or female between the ages of 2 and 50 years old. A parent or legal guardian must provide signed informed consent for participation.
  • The participant should be currently receiving stable standard treatments for at least 3 months before the first dose. This includes stable doses of medications for seizures, behavior, sleep, and other supportive treatments like special diets or supplements.
  • The participant must agree to follow good study practices, which include not sharing personal or study information on social media platforms like Facebook, Instagram, Twitter, or YouTube until the study is completed.
  • Other specific criteria defined by the study protocol may also apply.

Who cannot join the trial?

8 criteria

  • Patients who do not have a confirmed diagnosis of Angelman syndrome cannot participate. Angelman syndrome is a genetic disorder that affects the nervous system and causes developmental disabilities and neurological problems.
  • Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
  • Patients who are not able to safely undergo the study procedures, such as the administration of the study drug, cannot participate.
  • Patients who have other medical conditions that might interfere with the study or increase risk cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate, as the study may pose risks to the baby.
  • Patients who are currently participating in another clinical trial cannot participate, as this could affect the study results.
  • Patients who have a history of allergic reactions to similar medications cannot participate, to avoid potential health risks.
  • Patients who are unable to comply with the study requirements, such as attending scheduled visits, cannot participate.
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Investigated drugs

ION582 is a medication being studied for its safety and tolerability in patients with Angelman Syndrome. It is administered directly into the spinal fluid through a procedure called intrathecal injection. The study aims to understand how the body processes the medication and its effects on the body, focusing on any side effects or changes in health indicators.

What is already known about the treatment

ION582 – This medication is administered intrathecally, meaning it is injected directly into the spinal canal. It is currently being studied in clinical trials for its safety and effectiveness in treating Angelman syndrome, a rare genetic disorder. The main goal of the trial is to assess how well patients tolerate different doses and to monitor any side effects. ION582 works at the molecular level by targeting specific genetic pathways involved in Angelman syndrome. It is classified as an investigational drug, meaning it is still under research and not yet approved for general medical use.

Investigated diseases

Angelman syndrome – Angelman syndrome is a genetic disorder that primarily affects the nervous system. It is characterized by developmental delays, intellectual disability, severe speech impairment, and problems with movement and balance. Individuals with this condition often exhibit a happy demeanor, frequent smiling, and laughter. The syndrome is caused by a loss of function of a gene on chromosome 15. Symptoms typically become noticeable in early childhood, and while they do not worsen over time, they can affect various aspects of daily life. The condition is considered rare and is usually diagnosed based on clinical features and genetic testing.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-512589-32-00Protocol codeION582CS1Estimated enrolment59 patientsSponsorIonis Pharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).