Clinical trial NCT06415344: Long-term Extension of GTX-102 in Angelman Syndrome
Official title: A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Angelman Syndrome
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2024-05-16
Enrollment: 75
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: 5 Years
Secondary outcomes:
measure: Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score
timeFrame: Month 0, 5 Years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California, Los Angeles (UCLA)
state: California
city: San Diego
country: United States
facility: University of California, San Diego - Rady Children's Hospital
state: California
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: New York
country: United States
facility: Weill Cornell Medicine
state: New York
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Melbourne
country: Australia
facility: The Royal Children's Hospital
state: Victoria
city: Calgary
country: Canada
facility: MAGIC Clinic Ltd
state: Alberta
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: London
country: Canada
facility: Childrens Hospital London Health Sciences Centre
state: Ontario
city: Ottawa
country: Canada
facility: Childrens Hospital of Eastern Ontario
state: Ontario
city: Montreal
country: Canada
facility: The Research Institute of the McGill University Health Centre
state: Quebec
city: Marseille
country: France
facility: AP-HM - Hopital de la Timone
city: Paris
country: France
facility: AP-HP Hopital Necker-Enfants Malades
city: Hamburg
country: Germany
facility: Universitaetsklinikum Hamburg-Eppendorf
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta de Hierro
city: Sabadell
country: Spain
facility: Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
city: Cambridge
country: United Kingdom
facility: University of Cambridge
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust
Study officials:
affiliation: Ultragenyx Pharmaceuticals Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: