Long-term Extension of GTX-102 in Angelman Syndrome
- Registration Number
- NCT06415344
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- 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)
- Detailed 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.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 75
- 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.
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description GTX-102 GTX-102 Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.
- Primary Outcome Measures
Name Time Method Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) 5 Years
- Secondary Outcome Measures
Name Time Method Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score Month 0, 5 Years
Trial Locations
- Locations (23)
University of California, Los Angeles (UCLA)
🇺🇸Los Angeles, California, United States
University of California, San Diego - Rady Children's Hospital
🇺🇸San Diego, California, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Weill Cornell Medicine
🇺🇸New York, New York, United States
Queensland Children's Hospital
🇦🇺South Brisbane, Queensland, Australia
The Royal Children's Hospital
🇦🇺Melbourne, Victoria, Australia
MAGIC Clinic Ltd
🇨🇦Calgary, Alberta, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, British Columbia, Canada
Scroll for more (13 remaining)University of California, Los Angeles (UCLA)🇺🇸Los Angeles, California, United States