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GTX-102 for Angelman Syndrome (Aspire Trial)

Phase 3
Waitlist Available
Research Sponsored by Ultragenyx Pharmaceutical Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of Angelman Syndrome (AS) with genetic confirmation of full maternal UBE3A gene deletion causing AS in the region of 15q11.2 q13
Able to ambulate independently, or with assistance at the Screening Visit (note, a child whose primary means of mobility is by wheelchair is excluded from the study)
Must not have
Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit or any prior use of gene therapy or antisense oligonucleotide (ASO) regardless of duration since last administration
Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial

Summary

"This trial aims to see if GTX-102 can improve thinking and memory in people with Angelman Syndrome."

Who is the study for?
This trial is for children with Angelman Syndrome who can walk (with or without help) and have a specific genetic deletion. They must be able to handle anesthesia without needing a breathing tube, follow the study plan, and their blood clotting tests should be close to normal. Sexually active participants must use effective birth control.
What is being tested?
The trial is testing GTX-102's ability to improve cognitive function in kids with Angelman Syndrome compared to a fake procedure (Sham-LP). Parents or guardians give consent for participation and agree to follow all procedures including lumbar puncture.
What are the potential side effects?
Specific side effects of GTX-102 are not listed here, but may include reactions related to drug administration such as discomfort at injection site, potential allergic reactions, or other unforeseen complications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a confirmed diagnosis of Angelman Syndrome with a specific genetic mutation.
Select...
I can walk on my own or with help, but I do not use a wheelchair as my main way to move around.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I haven't used any experimental drugs or devices, gene therapy, or ASO recently.
Select...
I am taking or will take medication that could increase my risk of bleeding.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: GTX-102Experimental Treatment1 Intervention
Participants will receive GTX-102 via lumbar puncture (LP) during both the double-blind and open-label period
Group II: Sham-LP then GTX-102Placebo Group2 Interventions
Participants will receive sham procedure during the double-blind period and then will receive GTX-102 via LP during the open-label period

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Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor
93 Previous Clinical Trials
104,216 Total Patients Enrolled
3 Trials studying Angelman Syndrome
160 Patients Enrolled for Angelman Syndrome
Medical DirectorStudy DirectorUltragenyx Pharmaceuticals Inc.
2,887 Previous Clinical Trials
8,088,912 Total Patients Enrolled
3 Trials studying Angelman Syndrome
169 Patients Enrolled for Angelman Syndrome
~80 spots leftby Jul 2026
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