Long-term Extension of GTX-102 in Angelman Syndrome
About
Brief Summary
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
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
Join this Trial
Enrolling by Invitation
These studies are not open to everyone who meets the eligibility criteria, but
only to people in that particular population, who are specifically invited to participate.
Study Stats
Protocol No.
24-5192
Category
Genetic and Rare Diseases
Pediatric and Prenatal Disorders
Principal Investigator
Contact
Location
- UCLA Westwood