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The trial is taking place at:
R

Rare Disease Research, LLC | Atlanta, GA

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Long-term Extension of GTX-102 in Angelman Syndrome

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Ultragenyx

Status and phase

Begins enrollment this month
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)

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

Trial contacts and locations

24

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Central trial contact

HCPs Contact: Medical Information; Patients Contact: Trial Recruitment

Data sourced from clinicaltrials.gov

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