NCT06617429 (Aspire) is a Phase 3 clinical trial of GTX-102 in Angelman Syndrome, sponsored by Ultragenyx Pharmaceutical Inc.

Who is sponsoring NCT06617429?

NCT06617429 is sponsored by Ultragenyx Pharmaceutical Inc.

What drugs are being tested in NCT06617429?

Interventions in NCT06617429: GTX-102, Sham-LP.

What condition does NCT06617429 study?

NCT06617429 studies Angelman Syndrome.

Is NCT06617429 still recruiting?

NCT06617429 is currently active, enrolled. Last updated 4 May 2026.

Last reviewed 2026-05-04 · How we verify

NCT06617429: Aspire

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

A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome

Active, enrolled Phase 3 Last updated 4 May 2026

What this trial tests

Phase 3 trial testing GTX-102 in Angelman Syndrome in 129 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

3 December 2024

Primary endpoint
1 July 2026

1 November 2027

Quick facts

Lead sponsor	Ultragenyx Pharmaceutical Inc
Phase	Phase 3
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	129
Start date	3 December 2024
Primary completion	1 July 2026
Estimated completion	1 November 2027
Sites	28 locations across United States, Canada, Germany, Japan, Poland, Spain

Drugs / interventions tested

GTX-102 — full drug profile →
Sham-LP

Conditions studied

Angelman Syndrome — all drugs for Angelman Syndrome →

Sponsor

Ultragenyx Pharmaceutical Inc — full company profile →

Who can join

Adults 4 to 17, 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.

Change from Baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338
Time frame: Baseline, Day 338

Sponsor's own description

The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).

Publications & conference data

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

Novel therapeutics in autism spectrum disorder.
Sara Daniella Kevelson, Elmaghraby R, Patel F, Brown H, et al · · 2026 · cited 1× · PMID 41748402 · DOI 10.1016/j.neurot.2026.e00857
A dual-reporter mouse for therapeutic discovery in Angelman syndrome.
Vihma H, James LM, Nourie HC, Smith AL, et al · · 2026 · cited 1× · PMID 41632831 · DOI 10.1172/jci.insight.197028
Antisense Oligonucleotides: Technological Advances, Clinical Progress, and Expanding Therapeutic Frontiers.
Xu L, Zhang H, Jiang B, Jiang Y, et al · · 2026 · PMID 42076098 · DOI 10.3390/pharmaceutics18040446
Crossing the finish line towards a disease-modifying treatment for Angelman syndrome.
Judson MC, de Almeida LP, Burdine RD, Chamberlain SJ, et al · · 2026 · PMID 41795090 · DOI 10.1186/s11689-026-09681-5
Development of at-home video recordings for functional skill assessment in Angelman Syndrome: a pilot study.
Leffler M, Woods RJ, Sapp A, Zigler CK, et al · · 2026 · PMID 41714940 · DOI 10.1186/s11689-026-09676-2
Preliminary perspectives on gene therapy in fragile X syndrome: a caregiver view.
Eley SEA, Weissgold S, Stanfield AC. · · 2025 · PMID 40890602 · DOI 10.1186/s11689-025-09629-1

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 NCT06617429 (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/NCT06617429.

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