Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE3
255 participants
INTERVENTIONAL
2024-07-31
2029-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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GTX-102
Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.
GTX-102
Antisense Oligonucleotide
Interventions
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GTX-102
Antisense Oligonucleotide
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Completed a final study visit in a prior clinical trial with GTX-102. If the subject has an ongoing adverse event, medical monitor approval is required to rollover. The Screening visit must occur within 6 months of the last visit in the prior GTX-102 trial.
* 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
* History or evidence of any other medical, neurological, or psychological condition that would expose the subject to an undue risk of a significant AE or interfere with study assessments during the course of the trial as determined by the clinical judgment of the Investigator.
* 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
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceuticals Inc.
Locations
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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
Childrens Hospital London Health Sciences Centre
London, Ontario, Canada
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, Canada
The Research Institute of the McGill University Health Centre
Montreal, Quebec, Canada
AP-HM - Hopital de la Timone
Marseille, , France
AP-HP Hopital Necker-Enfants Malades
Paris, , France
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, , Germany
Universitaetsklinikum Leipzig
Leipzig, , Germany
Sheba Medical Center
Ramat Gan, , Israel
Hospital Universitario Puerta de Hierro
Majadahonda, , Spain
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell
Sabadell, , Spain
University of Cambridge
Cambridge, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford, , United Kingdom
Countries
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Related Links
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Ultragenyx Patient Advocacy Website
Ultragenyx Transparency Commitment
Ultragenyx Angelman Syndrome (AS) Research Study Opportunity
Other Identifiers
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2024-510917-14-00
Identifier Type: CTIS
Identifier Source: secondary_id
GTX-102-CL302
Identifier Type: -
Identifier Source: org_study_id
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