GTX-102 for Angelman Syndrome

Palo Alto (17 mi)

Age: Any Age

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Ultragenyx Pharmaceutical Inc

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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

Eligibility Criteria

This trial is for individuals with Angelman Syndrome who have previously participated in a GTX-102 study. They must consent to the trial's terms and agree to use effective contraception. Those pregnant, breastfeeding, or hypersensitive to GTX-102 or its components cannot join.

Inclusion Criteria

I have been in a GTX-102 study and got approval to join this one.

I am using or willing to use effective birth control or practice abstinence during and after the study.

Treatment Details

The study aims to assess the long-term safety of GTX-102 in patients with Angelman Syndrome. Participants are those who've been part of prior GTX-102 research and will continue treatment under careful monitoring.

1Treatment groups

Experimental Treatment

Group I: GTX-102Experimental Treatment1 Intervention

Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.