New hope for angelman syndrome: experimental drug enters Mid-Stage trial
NCT ID NCT07157254
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an experimental drug called GTX-102 (apazunersen) in 60 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants range from 1 to 65 years old and are grouped by age and genetic type. The goal is to see if the drug is safe and can improve skills like thinking, communication, and movement.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GTX-102 (apazunersen), an antisense oligonucleotide
What this could lead to
If successful, this could lead to a treatment that improves cognitive, communication, and motor skills in people with Angelman syndrome.
What could go wrong
This is an early Phase 2 study with only 60 participants, so results may not apply to everyone. The drug may cause side effects or fail to show meaningful improvement.
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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Akron Children's Hospital
RECRUITINGAkron, Ohio, 44308, United States
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Azienda Ospedaliera Universitaria Meyer IRCCS
RECRUITINGFlorence, Italy
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Carum Research Inc.
RECRUITINGDallas, Texas, 75243, United States
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Cedars Sinai Medical Center
RECRUITINGLos Angeles, California, 90048, United States
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Clinical Trial Site
NOT_YET_RECRUITINGBaltimore, Maryland, 21205, United States
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Clinical Trial Site
NOT_YET_RECRUITINGKansas City, Missouri, 64108, United States
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Clinical Trial Site
NOT_YET_RECRUITINGSanta Cecília, Porto Alegre, Brazil
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Clinical Trial Site
NOT_YET_RECRUITINGRome, Italy
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Clinical Trial Site
NOT_YET_RECRUITINGLondon, United Kingdom
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Fondazione IRCCS Istituto Neurologico C. Besta
RECRUITINGMilan, Italy
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Hopital de la Timone
RECRUITINGMarseille, France
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Hospital Santa Joao
RECRUITINGPorto, Portugal
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Hospital Universitario Austral
RECRUITINGPilar, Buenos Aires, Argentina
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Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe
RECRUITINGCuritiba, Paraná, Brazil
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Hospital de Santa Maria
RECRUITINGLisbon, Portugal
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Necker-Enfants Malades Hospital
RECRUITINGParis, France
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Rare Disease Research
RECRUITINGHillsborough, North Carolina, 27278, United States
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Rush University Medical Center
RECRUITINGChicago, Illinois, 60612, United States
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Sheba Medical Center
RECRUITINGRamat Gan, Israel
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UT Health Austin
RECRUITINGAustin, Texas, 78723, United States
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University of Oxford
RECRUITINGOxford, United Kingdom