This study is accepting new patients by invitation only

Long-term Extension of GTX-102 in Angelman Syndrome

a study on Angelman Syndrome

Summary

Location
at UCLA UCSD
Dates
study started
completion around

Description

Summary

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

Official Title

A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

Details

This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.

Keywords

Angelman Syndrome, Syndrome, GTX-102

Eligibility

You can join if…

  • Signed informed consent from parent(s) or legal guardian(s).
  • Prior participation in a clinical trial with GTX-102; the timing for the roll-over into this study from a prior GTX-102 study is based on the Investigator and Ultragenyx Medical Monitor's approval.
  • 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, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102.

You CAN'T join if...

  • Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects.
  • Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study

Locations

  • University of California, Los Angeles (UCLA)
    Los Angeles California 90095 United States
  • University of California, San Diego - Rady Children's Hospital
    San Diego California 92123 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Ultragenyx Pharmaceutical Inc
ID
NCT06415344
Phase
Phase 3 Angelman Syndrome Research Study
Study Type
Interventional
Participants
Expecting 75 study participants
Last Updated