- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06415344
Long-term Extension of GTX-102 in Angelman Syndrome
May 10, 2024 updated by: Ultragenyx Pharmaceutical Inc
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Study Overview
Detailed 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.
Study Type
Interventional
Enrollment (Estimated)
75
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Patients Contact: Trial Recruitment
- Phone Number: 1-888-756-8657
- Email: trialrecruitment@ultragenyx.com
Study Contact Backup
- Name: HCPs Contact: Medical Information
- Phone Number: 1-888-756-8657
- Email: medinfo@ultragenyx.com
Study Locations
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Queensland
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South Brisbane, Queensland, Australia, 4101
- Queensland Children's Hospital
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Victoria
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Heidelberg, Victoria, Australia, 3084
- Austin Health
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Melbourne, Victoria, Australia, 3052
- The Royal Children's Hospital
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Alberta
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Calgary, Alberta, Canada, T2E 7Z4
- MAGIC Clinic Ltd
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British Columbia
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Vancouver, British Columbia, Canada, V6H 3V4
- British Columbia Children's Hospital
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Ontario
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London, Ontario, Canada, N6A 5W9
- Childrens Hospital London Health Sciences Centre
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Ottawa, Ontario, Canada, K1H 8L1
- Childrens Hospital of Eastern Ontario
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Quebec
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Montreal, Quebec, Canada, H3G 1A4
- The Research Institute of the McGill University Health Centre
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Marseille, France, 13385
- AP-HM - Hôpital de la Timone
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Paris, France, 75015
- AP-HP Hopital Necker-Enfants Malades
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Hamburg, Germany, 20246
- Universitaetsklinikum Hamburg-Eppendorf
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Leipzig, Germany, 04103
- Universitaetsklinikum Leipzig
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Tel HaShomer, Israel, 5265601
- Sheba Medical Center
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Majadahonda, Spain, 28222
- Hospital Universitario Puerta de Hierro
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Sabadell, Spain, 08208
- Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
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Cambridge, United Kingdom, CB2 0QQ
- University of Cambridge
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London, United Kingdom, WC1N 3JH
- Great Ormond Street Hospital for Children
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Oxford, United Kingdom, OX3 9DU
- Oxford University Hospitals Nhs Foundation Trust
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California
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Los Angeles, California, United States, 90095
- University of California, Los Angeles (UCLA)
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San Diego, California, United States, 92123
- University of California, San Diego - Rady Children's Hospital
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Georgia
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Atlanta, Georgia, United States, 30329
- Rare Disease Research, LLC
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Illinois
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Chicago, Illinois, United States, 60612
- Rush University Medical Center
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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New York
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New York, New York, United States, 10021
- Weill Cornell Medicine
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: GTX-102
Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.
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Antisense Oligonucleotide
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: 5 Years
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5 Years
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Change from LTE Month 0 and Pretreatment in the Bayley-4 Raw Score
Time Frame: Month 0, 5 Years
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Month 0, 5 Years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Ultragenyx Pharmaceuticals Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Estimated)
August 1, 2024
Primary Completion (Estimated)
February 1, 2029
Study Completion (Estimated)
February 1, 2029
Study Registration Dates
First Submitted
May 10, 2024
First Submitted That Met QC Criteria
May 10, 2024
First Posted (Actual)
May 16, 2024
Study Record Updates
Last Update Posted (Actual)
May 16, 2024
Last Update Submitted That Met QC Criteria
May 10, 2024
Last Verified
May 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- GTX-102-CL302
- 2024-510917-14-00 (Ctis)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Angelman Syndrome
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Ovid Therapeutics Inc.CompletedPrimary Disease or Condition Being Studied: Angelman Syndrome (AS)United States, Australia, Germany, Israel, Netherlands
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University of OxfordHoffmann-La Roche; Foundation for Angelman Syndrome Therapeutics UKRecruiting
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Fondazione per la Ricerca Ospedale MaggioreCompleted
-
Ovid Therapeutics Inc.CompletedAngelman SyndromeUnited States, Israel
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Boston Children's HospitalCompletedAngelman SyndromeUnited States
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Puerta de Hierro University HospitalParc Taulí Hospital UniversitariRecruiting
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Foundation for Angelman Syndrome Therapeutics,...Queensland University of TechnologyRecruiting
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The Emmes Company, LLCBoston Children's Hospital; Ionis Pharmaceuticals, Inc.CompletedAngelman SyndromeUnited States
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Hoffmann-La RocheActive, not recruitingAngelman SyndromeUnited States, Spain, Netherlands, Italy
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-
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-
Amicus TherapeuticsActive, not recruiting
-
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-
GTxCompletedProstate CancerUnited States
-
GTxWithdrawnStress Urinary IncontinenceUnited States
-
GTxCompletedStress Urinary IncontinenceUnited States
-
GTxWithdrawnStress Urinary IncontinenceUnited States
-
GTxCompletedStress Urinary IncontinenceUnited States