Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome Clinical Trial

Official title:

A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06415344
• Other study ID #: GTX-102-CL302
• Secondary ID: 2024-510917-14-0
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: August 2024
• Est. completion date: February 2029

Study information

• Verified date: May 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: Patients Contact: Trial Recruitment
• Phone: 1-888-756-8657
• Email: trialrecruitment[@]ultragenyx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 75
• Est. completion date: February 2029
• Est. primary completion date: February 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Angelman Syndrome
• Syndrome

Intervention

Drug:
• GTX-102
Antisense Oligonucleotide

Locations

Country	Name	City	State
Australia	Austin Health	Heidelberg	Victoria
Australia	The Royal Children's Hospital	Melbourne	Victoria
Australia	Queensland Children's Hospital	South Brisbane	Queensland
Canada	MAGIC Clinic Ltd	Calgary	Alberta
Canada	Childrens Hospital London Health Sciences Centre	London	Ontario
Canada	The Research Institute of the McGill University Health Centre	Montreal	Quebec
Canada	Childrens Hospital of Eastern Ontario	Ottawa	Ontario
Canada	British Columbia Children's Hospital	Vancouver	British Columbia
France	AP-HM - Hopital de la Timone	Marseille
France	AP-HP Hopital Necker-Enfants Malades	Paris
Germany	Universitaetsklinikum Hamburg-Eppendorf	Hamburg
Germany	Universitaetsklinikum Leipzig	Leipzig
Israel	Sheba Medical Center	Tel HaShomer
Spain	Hospital Universitario Puerta de Hierro	Majadahonda
Spain	Corporacio Sanitaria Parc Tauli - Hospital de Sabadell	Sabadell
United Kingdom	University of Cambridge	Cambridge
United Kingdom	Great Ormond Street Hospital for Children	London
United Kingdom	Oxford University Hospitals NHS Foundation Trust	Oxford
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	University of California, Los Angeles (UCLA)	Los Angeles	California
United States	Weill Cornell Medicine	New York	New York
United States	University of California, San Diego - Rady Children's Hospital	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany,  Israel,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)		5 Years
Secondary	Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score		Month 0, 5 Years