New hope for angelman syndrome: experimental drug ION582 enters human trials

NCT ID NCT05127226

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

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.

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.

Angelman syndrome

As listed by the trial registrant

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

ANGELMAN SYNDROME

Contacts and locations

Locations

Azienda Ospedaliera Universitaria Pisana
RECRUITING

Pisa, 56126, Italy
Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02215, United States
Colorado Children's Hospital Research Institute
RECRUITING

Aurora, Colorado, 80045, United States
Necker-Enfants Malades Hospital
RECRUITING

Paris, 75015, France
Rady Children's Hospital
RECRUITING

San Diego, California, 92123, United States
Rush University Medical Center
RECRUITING

Chicago, Illinois, 60612, United States
STRONG Group University of Oxford
ACTIVE_NOT_RECRUITING

Oxford, Oxfordshire, OX3 9DU, United Kingdom
Sheba Medical Center
RECRUITING

Ramat Gan, 5262100, Israel
Sydney Children's Hospital, Kids Cancer Centre
ACTIVE_NOT_RECRUITING

Randwick, NSW 2031, Australia
Texas Children's Hospital
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
University of North Carolina at Chapel Hill School of Medicine
RECRUITING

Carrboro, North Carolina, 27510, United States

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