GTX-102 for Angelman Syndrome
This study is testing a drug called GTX-102 for people with Angelman syndrome. Researchers want to see how safe and effective GTX-102 is. Participants can be between 1 and 64 years old and have a confirmed diagnosis of Angelman syndrome, specifically the deletion-type for some groups. The study will measure side effects and look at how GTX-102 affects cognitive (thinking and learning) abilities and overall response to treatment. The study is currently unclear on its recruitment status, but plans to enroll 60 participants. Some participants will receive GTX-102, while others in one group will not receive treatment for a period.
- Study design
- This study is an interventional basket study with several subprotocols. Most subprotocols are open-label (everyone knows what treatment is being given) and single-arm, while one subprotocol randomizes participants 2:1 to receive GTX-102 or no treatment.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for safety up to Day 506. Cognitive and multidomain responses will be measured at Day 338.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
At a glance
Conditions
Where it's being run
21 sites across 15 statesStudy leadership
- Medical Director · STUDY_DIRECTOR · Ultragenyx Pharmaceutical Inc
Who to contact
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What this trial measures
- 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 PremedicationUp to Day 506
- Subprotocol A Only: Bayley-4 Cognitive Without Caregiver Input Raw Score Change from Baseline at Day 338Baseline, Day 338
- Subprotocol B/D Only: Multidomain Responder Index (MDRI) Net Response at Day 338Baseline, 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 accordingly, and a summary measure of net response will then be calculated across all participants.
- Subprotocol C Only: MDRI Net Response at Day 338Baseline, 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.