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Gene Therapy

GTX-102 for Angelman Syndrome

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
Prior participation in a clinical trial with GTX-102 with approval from the Investigator and Ultragenyx Medical Monitor
Females of childbearing potential who are sexually active must use highly effective contraception or abstinence from the time of informed consent through the end of the study and for at least 6 months after the final dose of GTX-102. Males must agree to remain abstinent or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights

Summary

"This trial is looking at the long-term safety of GTX-102 in people with Angelman Syndrome."

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects are not listed here, they would typically include any adverse reactions participants have had during previous trials with GTX-102, as well as potential new long-term safety concerns.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been in a GTX-102 study and got approval to join this one.
Select...
I am using or willing to use effective birth control or practice abstinence during and after the study.

Timeline

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score

Trial Design

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.

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Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor
90 Previous Clinical Trials
179,040 Total Patients Enrolled
2 Trials studying Angelman Syndrome
85 Patients Enrolled for Angelman Syndrome
Medical DirectorStudy DirectorUltragenyx Pharmaceuticals Inc.
2,801 Previous Clinical Trials
8,070,255 Total Patients Enrolled
2 Trials studying Angelman Syndrome
94 Patients Enrolled for Angelman Syndrome
~50 spots leftby Feb 2029
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