RecruitingPhase 2NCT07157254

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome

Sponsor

Ultragenyx Pharmaceutical Inc

Enrollment

60 participants

Start Date

Oct 13, 2025

Study Type

INTERVENTIONAL

Conditions

Angelman Syndrome

Summary

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Eligibility

Min Age: 1 YearMax Age: 64 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called GTX-102 and No intervention for people with angelman syndrome. The study is currently recruiting participants at 21 locations. People eligible for this study include aged 1 Year to 64 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERNo intervention

During the no treatment period participants do not receive any study drug

DRUGGTX-102

antisense oligonucleotide

Locations(21)

Cedars Sinai Medical Center

Los Angeles, California, United States

Rush University Medical Center

Chicago, Illinois, United States

Clinical Trial Site

Baltimore, Maryland, United States

Clinical Trial Site

Kansas City, Missouri, United States

Rare Disease Research

Hillsborough, North Carolina, United States

Akron Children's Hospital

Akron, Ohio, United States

UT Health Austin

Austin, Texas, United States

Carum Research Inc.

Dallas, Texas, United States

Hospital Universitario Austral

Pilar, Buenos Aires, Argentina

Hospital de Crianças César Pernetta e Hospital Pequeno Príncipe

Curitiba, Paraná, Brazil

Clinical Trial Site

Santa Cecília, Porto Alegre, Brazil

Hopital de la Timone

Marseille, France

Necker-Enfants Malades Hospital

Paris, France

Sheba Medical Center

Ramat Gan, Israel

Azienda Ospedaliera Universitaria Meyer IRCCS

Florence, Italy

Fondazione IRCCS Istituto Neurologico C. Besta

Milan, Italy

Clinical Trial Site

Rome, Italy

Hospital de Santa Maria

Lisbon, Portugal

Hospital Santa Joao

Porto, Portugal

Clinical Trial Site

London, United Kingdom

University of Oxford

Oxford, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT07157254

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