Long-term Extension of GTX-102 in Angelman Syndrome

Ultragenyx

Status and phase

Invitation-only

Phase 3

Conditions

Angelman Syndrome

Treatments

Drug: GTX-102

Study type

Interventional

Funder types

Industry

Identifiers

NCT06415344

GTX-102-CL302

2024-510917-14-00

Details and patient eligibility

About

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

Full description

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.

Enrollment

75 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

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