New drug aims to unlock speech in angelman syndrome
NCT ID NCT06914609
First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 4 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Associazione La Nostra Famiglia - IRCCS Eugenio Medea
NOT_YET_RECRUITINGConegliano, 31015, Italy
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Austin Hospital
RECRUITINGHeidelberg, 3084, Australia
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Azienda Ospedaliero Universitaria Pisana
RECRUITINGPisa, 56126, Italy
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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British Columbia Children's Hospital
RECRUITINGVancouver, V6H 3V4, Canada
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Cedars-Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
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Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Mercy
RECRUITINGKansas City, Missouri, 64108, United States
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20011, United States
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Cincinnati Children's Hospital
RECRUITINGCincinnati, Ohio, 45229, United States
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Colorado Children's Hospital Research Institute
RECRUITINGAurora, Colorado, 80045, United States
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Columbia University Irving Medical Center
RECRUITINGNew York, New York, 10032, United States
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Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
RECRUITINGSabadell, 08208, Spain
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Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITINGMilan, 20133, Italy
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Great Ormond Street Hospital for Children - NHS Foundation Trust
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Sant Joan de Deu
RECRUITINGBarcelona, 08950, Spain
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Ichan School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
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John Radcliffe Hospital
RECRUITINGOxford, OX3 9DU, United Kingdom
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KK Women's and Children's Hospital
RECRUITINGSingapore, 229899, Singapore
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Klinikum der Ludwig-Maximilians-Universitaet Muenchen
RECRUITINGMünchen, 81377, Germany
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London Health Science Centre - Children's Hospital
RECRUITINGLondon, Ontario, N6A 5W9, Canada
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McGill University Health Centre
RECRUITINGMontreal, Quebec, H4A 3J1, Canada
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National Center of Neurology and Psychiatry
RECRUITINGKodaira, Tokyo, 187-8551, Japan
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Nicklaus Children's Hospital
RECRUITINGMiami, Florida, 33155, United States
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Osaka Women's and Children's Hospital
RECRUITINGIzumi, Osaka, 594-1101, Japan
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Ospedale Pediatrico Bambino Gesù
RECRUITINGRoma, 00165, Italy
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Perth Children's Hospital
RECRUITINGNedlands, 6009, Australia
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Queensland Children's Hospital
RECRUITINGSouth Brisbane, Queensland, 4101, Australia
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Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
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Samsung Medical Center
RECRUITINGSeoul, 06351, South Korea
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Seoul National University Hospital
RECRUITINGSeoul, 03080, South Korea
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Sheba Medical Center
RECRUITINGRamat Gan, 5265601, Israel
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Sydney Children's Hospital
RECRUITINGRandwick, 2031, Australia
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
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UCLA Clinical & Translational Research Center (CTRC)
RECRUITINGLos Angeles, California, 90095, United States
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University of Alberta Hospital
RECRUITINGEdmonton, T6G 2B7, Canada
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University of North Carolina at Chapel Hill School of Medicine
RECRUITINGCarrboro, North Carolina, 27510, United States
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Uniwersyteckie Centrum Kliniczne
RECRUITINGGdansk, 80-952, Poland
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Vanderbilt Clinical Research Center
RECRUITINGNashville, Tennessee, 37232, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can early parent coaching help infants with rare genetic disorders thrive?
- Newborn screening study aims to catch rare diseases at birth
- New hope for angelman syndrome: drug trial targets brain function
- New registry aims to shed light on angelman syndrome
- New study tracks angelman syndrome progression in kids and adults
- 2000-Patient study aims to uncover hidden metabolic risks in rare genetic disorders