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

U.S., Sept. 6 -- ClinicalTrials.gov registry received information related to the study (NCT07157254) titled 'A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)' on Aug. 21.

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

Study Start Date: Nov., 2025

Study Type: INTERVENTIONAL

Condition: Angelman Syndrome

Intervention: OTHER: No intervention

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

DRUG: GTX-102

antisense oligonucleotide

Recruitment Status: NOT_YET_RECRUITING

Sponsor: Ultragenyx Pharmaceutical Inc

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