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 is a Phase 3 long term extension (LTE) study to evaluate the safety and efficacy of GTX-102 in participants with AS.

Participants may remain in the study until GTX-102 is approved and/or becomes available in their geographical region or the sponsor stops the study.

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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75 participants in 1 patient group

GTX-102

Experimental group

Description:

Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.

Treatment:

Drug: GTX-102