NCT06415344 is a Phase 3 clinical trial of GTX-102 in Angelman Syndrome, sponsored by Ultragenyx Pharmaceutical Inc.

Who is sponsoring NCT06415344?

NCT06415344 is sponsored by Ultragenyx Pharmaceutical Inc.

What drugs are being tested in NCT06415344?

Interventions in NCT06415344: GTX-102.

What condition does NCT06415344 study?

NCT06415344 studies Angelman Syndrome.

Is NCT06415344 still recruiting?

NCT06415344 is currently enrolling by invitation. Last updated 4 May 2026.

Last reviewed 2026-05-04 · How we verify

NCT06415344

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

ENROLLING BY INVITATION Phase 3 Last updated 4 May 2026

What this trial tests

Phase 3 trial testing GTX-102 in Angelman Syndrome in 255 participants. Enrolling by invitation.

Timeline

31 July 2024

Primary endpoint
1 February 2029

1 February 2029

Quick facts

Lead sponsor	Ultragenyx Pharmaceutical Inc
Phase	Phase 3
Status	ENROLLING BY INVITATION
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	255
Start date	31 July 2024
Primary completion	1 February 2029
Estimated completion	1 February 2029
Sites	23 locations across United States, Australia, Canada, France, Germany, Israel, Spain, United Kingdom

Drugs / interventions tested

GTX-102 — full drug profile →

Conditions studied

Angelman Syndrome — all drugs for Angelman Syndrome →

Sponsor

Ultragenyx Pharmaceutical Inc — full company profile →

Who can join

Eligibility, any sex, with Angelman Syndrome. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 5 Years

Sponsor's own description

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

Antisense oligonucleotides and their applications in rare neurological diseases.
McDowall S, Aung-Htut M, Wilton S, Li D. · · 2024 · cited 10× · PMID 39376536 · DOI 10.3389/fnins.2024.1414658
A stem cell-based toolkit to model Angelman syndrome caused by paternal uniparental disomy of chromosome 15.
Cazaux Mateus F, Camões Dos Santos J, Arez M, Bekman EP, et al · · 2025 · PMID 40956516 · DOI 10.1007/s13577-025-01287-8

Verify or expand the search:

Other trials of GTX-102

Trials testing the same drug.

NCT04259281 — A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome · Phase 1, PHASE2 · completed

Other recruiting trials for Angelman Syndrome

Currently open trials in the same condition.

NCT07181837 — A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome · Phase 1, PHASE2 · recruiting
NCT06353620 — Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy · recruiting
NCT06115109 — Angelman Natural History Study - FAST Spain · recruiting
NCT05293184 — The Global Angelman Syndrome Registry · recruiting

Other Ultragenyx Pharmaceutical Inc trials

Trials by the same sponsor.

NCT04812106 — Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program · terminated
NCT05196165 — Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storag · terminated
NCT05312697 — Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta · Phase 2 · terminated
NCT04783428 — Tumor-induced Osteomalacia Disease Monitoring Program · active not recruiting
NCT05139316 — A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen S · Phase 3 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT06415344 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Ultragenyx Pharmaceutical Inc
Last refreshed: 4 May 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06415344.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing