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New drug aims to unlock speech in angelman syndrome

NCT ID NCT06914609

Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 5 times

Summary

This Phase 3 study tests an experimental drug called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The drug is given as a spinal injection and aims to improve expressive communication and cognitive skills. Participants will be randomly assigned to receive either ION582 or a placebo, and researchers will measure changes in communication abilities over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ION582
What this could lead to
If successful, this could lead to a treatment that improves communication and cognitive skills in people with Angelman syndrome.
What could go wrong
This is still an experimental drug. It may not work better than placebo, and side effects from the spinal injection or the drug itself are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 158 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Apr 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 50 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. The participants caregiver(s)/ legally authorized representative must have given written informed consent and any authorizations required by local law and be able to comply with all study requirements. 2. Medically stable and can undergo sedation and/or general anesthesia without intubation. 3. Male or female between 2 and lesser than or equal to (≤)50 years of age, depending on specific cohort, at the time of the in-clinic Screening visit. 4. Participant has a clinical diagnosis of Angelman syndrome (AS) with molecular confirmation of either Ubiquitin-protein ligase E3A (UBE3A) deletion or UBE3A mutation. 5. Currently receiving stable doses of concomitant medications typically prescribed for AS, such as anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and special diets, supplements, or nutritional support for at least 8 weeks prior to the Baseline visit. 6. Legally authorized representative/caregiver(s) agree(s) not to post any of the participant's personal medical data or information related to the study on any website or social media site (e.g., Facebook, Instagram, X (formerly Twitter), YouTube, TikTok, etc.) from the time of enrollment until they are notified that the study is completed. Key Exclusion Criteria: 1. Must not have any clinically significant abnormalities in medical history (e.g., major surgery within 3 months of screening), or on physical examination for which treatment with an antisense oligonucleotide (ASO) would be contraindicated or which, in the opinion of the Principal Investigator (PI), could confound the results of this study. 2. Known brain or spinal disease that would interfere with the lumbar puncture (LP) procedure, cerebrospinal fluid (CSF) circulation, or presence of other factors would affect the safety of the LP procedure. 3. Must not have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion or could interfere with the participant participating in or completing the study. 4. Must not have any laboratory abnormalities or any other clinically significant abnormalities that would, as assessed by the Investigator, at screening or Baseline, render a participant unsuitable for inclusion. 5. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid (RNA) \[siRNA\], ASOs) gene therapy or gene editing. This exclusion criterion does not apply to approved nucleic acid-based vaccines, including mRNA vaccines, which are allowed. 6. Has molecular confirmation of AS due to paternal uniparental disomy, imprinting center defect, or mosaic findings. Other inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

Angelman syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Angelman syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    41 sites in 12 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Austin Hospital

    RECRUITING

    Heidelberg, 3084, Australia

  • Azienda Ospedaliero Universitaria Pisana

    RECRUITING

    Pisa, 56126, Italy

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • British Columbia Children's Hospital

    RECRUITING

    Vancouver, V6H 3V4, Canada

  • Cedars-Sinai Medical Center

    RECRUITING

    Los Angeles, California, 90048, United States

  • Children's Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Mercy

    RECRUITING

    Kansas City, Missouri, 64108, United States

  • Children's National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20011, United States

  • Cincinnati Children's Hospital

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Colorado Children's Hospital Research Institute

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Corporacio Sanitaria Parc Tauli - Hospital de Sabadell

    RECRUITING

    Sabadell, 08208, Spain

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

  • Great Ormond Street Hospital for Children - NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, 08950, Spain

  • Ichan School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

  • John Radcliffe Hospital

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

  • KK Women's and Children's Hospital

    RECRUITING

    Singapore, 229899, Singapore

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    RECRUITING

    München, 81377, Germany

  • London Health Science Centre - Children's Hospital

    RECRUITING

    London, Ontario, N6A 5W9, Canada

  • McGill University Health Centre

    RECRUITING

    Montreal, Quebec, H4A 3J1, Canada

  • National Center of Neurology and Psychiatry

    RECRUITING

    Kodaira, Tokyo, 187-8551, Japan

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

  • Osaka Women's and Children's Hospital

    RECRUITING

    Izumi, Osaka, 594-1101, Japan

  • Ospedale Pediatrico Bambino Gesù

    RECRUITING

    Roma, 00165, Italy

  • Perth Children's Hospital

    RECRUITING

    Nedlands, 6009, Australia

  • Queensland Children's Hospital

    RECRUITING

    South Brisbane, Queensland, 4101, Australia

  • Rady Children's Hospital

    RECRUITING

    San Diego, California, 92123, United States

  • Rush University Medical Center

    RECRUITING

    Chicago, Illinois, 60612, United States

  • Samsung Medical Center

    RECRUITING

    Seoul, 06351, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Sheba Medical Center

    RECRUITING

    Ramat Gan, 5265601, Israel

  • Sydney Children's Hospital

    RECRUITING

    Randwick, 2031, Australia

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • UCLA Clinical & Translational Research Center (CTRC)

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of Alberta Hospital

    RECRUITING

    Edmonton, T6G 2B7, Canada

  • University of North Carolina at Chapel Hill School of Medicine

    RECRUITING

    Carrboro, North Carolina, 27510, United States

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gdansk, 80-952, Poland

  • Vanderbilt Clinical Research Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.