NCT07157254: Aurora

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

Recruiting now Phase 2 Last updated 3 June 2026

What this trial tests

Phase 2 trial testing No intervention in Angelman Syndrome in 60 participants. Currently enrolling.

Timeline

13 October 2025

Primary endpoint
1 January 2030

1 January 2030

Quick facts

Lead sponsor	Ultragenyx Pharmaceutical Inc
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	60
Start date	13 October 2025
Primary completion	1 January 2030
Estimated completion	1 January 2030
Sites	21 locations across United States, Argentina, Brazil, France, Israel, Italy, Portugal, United Kingdom

Drugs / interventions tested

• No intervention
• GTX-102 — full drug profile →

Conditions studied

• Angelman Syndrome — all drugs for Angelman Syndrome →

Sponsor

Ultragenyx Pharmaceutical Inc — full company profile →

Who can join

Adults 1 to 64, 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.

• Subprotocol A/B/C/D: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), Severe Events, and Events Related to Investigational Product, Procedure, and Premedication
  Time frame: Up to Day 506
• Subprotocol A Only: Bayley-4 Cognitive Without Caregiver Input Raw Score Change from Baseline at Day 338
  Time frame: Baseline, Day 338
• Subprotocol B/D Only: Multidomain Responder Index (MDRI) Net Response at Day 338
  Time frame: Baseline, Day 338
  The following assessments will be included to calculate the MDRI net response: Bayley-4 Cognitive and Receptive Communication, Aberrant Behavior Checklist- Community (ABC-C) Hyperactivity/Noncompliance (H/N), Angelman Severity Assessment (ASA) Sleep, ASA Gross Motor. For each assessment a meaningful score difference (MSD) is defined. A single net response score per participant will be derived acco
• Subprotocol C Only: MDRI Net Response at Day 338
  Time frame: Baseline, Day 338
  The following assessments will be included to calculate the MDRI net response: Vineland-3 Expressive and Receptive Communication, ABC-C Irritability, ASA Gross Motor. For each assessment a meaningful score difference (MSD) is defined. A single net response score per participant will be derived accordingly, and a summary measure of net response will then be calculated across all participants.

Sponsor's own description

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

Publications & conference data

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

1. 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
2. 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