Long-term Extension of GTX-102 in Angelman Syndrome

Launched by ULTRAGENYX PHARMACEUTICAL INC · May 10, 2024

Keywords

ClinConnect Summary

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.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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.
• Exclusion Criteria:
• • 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