New hope for angelman syndrome: experimental drug enters Mid-Stage trial

NCT ID NCT07157254

Recruiting now Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 3 times

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.

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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

Email: •••••@•••••

Locations

Akron Children's Hospital
RECRUITING

Akron, Ohio, 44308, United States
Azienda Ospedaliera Universitaria Meyer IRCCS
RECRUITING

Florence, Italy
Carum Research Inc.
RECRUITING

Dallas, Texas, 75243, United States
Casa dos Raros
RECRUITING

Santa Cecília, Porto Alegre, Brazil
Cedars Sinai Medical Center
RECRUITING

Los Angeles, California, 90048, United States
Clinical Trial Site
NOT_YET_RECRUITING

Kansas City, Missouri, 64108, United States
Clinical Trial Site
NOT_YET_RECRUITING

Rome, Italy
Clinical Trial Site
NOT_YET_RECRUITING

London, United Kingdom
Fondazione IRCCS Istituto Neurologico C. Besta
RECRUITING

Milan, Italy
Hopital de la Timone
RECRUITING

Marseille, France
Hospital Santa Joao
RECRUITING

Porto, Portugal
Hospital Universitario Austral
RECRUITING

Pilar, Buenos Aires, Argentina
Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe
RECRUITING

Curitiba, Paraná, Brazil
Hospital de Santa Maria
RECRUITING

Lisbon, Portugal
Kennedy Kreiger Institute
RECRUITING

Baltimore, Maryland, 21205, United States
Necker-Enfants Malades Hospital
RECRUITING

Paris, France
Rare Disease Research
RECRUITING

Hillsborough, North Carolina, 27278, United States
Rush University Medical Center
RECRUITING

Chicago, Illinois, 60612, United States
Sheba Medical Center
RECRUITING

Ramat Gan, Israel
UT Health Austin
RECRUITING

Austin, Texas, 78723, United States
University of Oxford
RECRUITING

Oxford, United Kingdom

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