New hope for angelman syndrome: experimental drug ION582 enters human trials
NCT ID NCT05127226
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called ION582 for people with Angelman syndrome, a genetic condition causing developmental delays and seizures. The drug is given via a spinal injection. The main goal is to check safety and how the body processes the drug. Up to 70 participants aged 0 to 50 will take part.
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 point toward a treatment that improves symptoms and quality of life for people with Angelman syndrome.
What could go wrong
This is an early-phase trial with only 70 participants, so safety and effectiveness are not yet proven. The drug is given via spinal injection, which carries risks like headache or infection.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Azienda Ospedaliera Universitaria Pisana
RECRUITINGPisa, 56126, Italy
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02215, United States
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Colorado Children's Hospital Research Institute
RECRUITINGAurora, Colorado, 80045, United States
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Necker-Enfants Malades Hospital
RECRUITINGParis, 75015, France
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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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STRONG Group University of Oxford
ACTIVE_NOT_RECRUITINGOxford, Oxfordshire, OX3 9DU, United Kingdom
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Sheba Medical Center
RECRUITINGRamat Gan, 5262100, Israel
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Sydney Children's Hospital, Kids Cancer Centre
ACTIVE_NOT_RECRUITINGRandwick, NSW 2031, Australia
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Texas Children's Hospital
ACTIVE_NOT_RECRUITINGHouston, Texas, 77030, United States
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University of North Carolina at Chapel Hill School of Medicine
RECRUITINGCarrboro, North Carolina, 27510, United States