New drug aims to unlock speech in angelman syndrome

NCT ID NCT06914609

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Associazione La Nostra Famiglia - IRCCS Eugenio Medea
NOT_YET_RECRUITING

Conegliano, 31015, Italy
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
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