Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT ID: NCT06617429
Last Updated: 2025-11-06
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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ACTIVE_NOT_RECRUITING
PHASE3
129 participants
INTERVENTIONAL
2024-12-03
2027-11-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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GTX-102
Participants will receive GTX-102 via lumbar puncture (LP) during both the double-blind and open-label period
GTX-102
antisense oligonucleotide
Sham-LP then GTX-102
Participants will receive sham procedure during the double-blind period and then will receive GTX-102 via LP during the open-label period
Sham-LP
Small needle prick on the lower back at the location where the LP injection is normally made
GTX-102
antisense oligonucleotide
Interventions
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Sham-LP
Small needle prick on the lower back at the location where the LP injection is normally made
GTX-102
antisense oligonucleotide
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of AS with genetic confirmation of full maternal ubiquitin-protein ligase E3A (UBE3A) gene deletion causing AS in the region of 15q11.2 q13
* Able to ambulate independently, or with assistance at the Screening Visit (note, a child whose primary means of mobility is by wheelchair is excluded from the study)
* Platelet count, prothrombin time / international normalized ratio, and partial thromboplastin time within 1.5x the normal limits at the Screening Visit
* Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including LP procedure, MRI, and tolerating anesthesia without intubation
* From the time of informed consent through to 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
* Any condition that creates an increased risk of unsuccessful LP
* Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
* Known hypersensitivity to GTX-102 or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
* Presence or history of any condition, lab abnormality, or infection, that, in the judgement of the Investigator, would interfere with participation, pose undue safety risk, or would confound interpretation of results
* Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
* Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit or any prior use of gene therapy or ASO regardless of duration since last administration
* Concurrent participation in any interventional study
4 Years
17 Years
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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Cedars Sinai
Los Angeles, California, United States
UCSD, Rady Children's Hospital
San Diego, California, United States
UCSF
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
Rush University
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Mercy
Kansas City, Missouri, United States
Columbia University Medical Center
New York, New York, United States
UNC Chapel Hill Pediatrics
Chapel Hill, North Carolina, United States
Rare Disease Research
Hillsborough, North Carolina, United States
The University of Texas
Austin, Texas, United States
Carum Research Inc
Dallas, Texas, United States
McGill University Health Centre
Montreal, Quebec, Canada
British Columbia Children's Hospital
Vancouver, , Canada
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, , Germany
University of Leipzig
Leipzig, , Germany
Haunersche Kinderklinik
Munich, , Germany
Nagoya City University Graduate School of Medical Sciences
Nagoya, Aichi-ken, Japan
Osaka City General Hospital
Osaka, , Japan
Hokkaido University Hospital
Sapporo, , Japan
Medical University of Gdańsk
Gdansk, , Poland
Polish Mothers Memorial Institute
Lodz, , Poland
Hospital Universitario Parc Tauli
Barcelona, , Spain
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitario Puerta de Hierro
Madrid, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Countries
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Related Links
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Ultragenyx Patient Advocacy Website
Ultragenyx Angelman Syndrome (AS) Research Study Opportunity
Ultragenyx Transparency Commitment
Other Identifiers
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2024-512600-19-00
Identifier Type: CTIS
Identifier Source: secondary_id
GTX-102-CL301
Identifier Type: -
Identifier Source: org_study_id