Actively Recruiting
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Led by Ultragenyx Pharmaceutical Inc · Updated on 2026-04-24
60
Participants Needed
22
Research Sites
220 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
CONDITIONS
Official Title
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent from parent(s) or legal guardian(s)
- Males and females aged 1 to under 4 years with deletion-type Angelman syndrome (Subprotocol A)
- Males and females aged 4 to under 18 years with UPD/ICD Angelman syndrome (Subprotocol B)
- Males and females aged 18 to under 65 years with any genotype of Angelman syndrome (Subprotocol C)
- Males and females aged 4 to under 18 years with mutation-type Angelman syndrome (Subprotocol D)
- Weight of at least 8 kg at screening
- Platelet count, prothrombin time/international normalized ratio, and partial thromboplastin time less than 1.5 times the upper limit of normal and platelets greater than 75,000 cells/mm3 at screening
- Willing and able to follow scheduled visits, study drug plan, laboratory tests, lumbar puncture, MRI, and tolerate anesthesia without intubation
- Females of childbearing potential who are sexually active must use highly effective contraception or abstain from sex during the study and for at least 6 months after the final dose
- Males must agree to abstain from heterosexual intercourse or use acceptable contraception during the study and for at least 3 months after the final dose
You will not qualify if you...
- Changes in medications or diet/supplements to treat Angelman syndrome symptoms within 1 month before screening (except weight-based adjustments)
- Conditions increasing risk of unsuccessful lumbar puncture
- Current or expected use of drugs increasing bleeding risk (e.g., heparin, platelet inhibitors)
- Known allergy to GTX-102 or its components or required premedication posing increased risk
- Any condition, lab abnormality, or infection that would interfere with study participation or safety
- Pregnant, breastfeeding, or planning pregnancy during the study (self or partner)
- Use of investigational products or medical devices within 6 months or 5 half-lives before screening, or any prior gene therapy or antisense oligonucleotide use
- Participation in any other interventional study concurrently
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 22 locations
1
Cedars Sinai Medical Center
Los Angeles, California, United States, 90048
Actively Recruiting
2
Rush University Medical Center
Chicago, Illinois, United States, 60612
Actively Recruiting
3
Clinical Trial Site
Baltimore, Maryland, United States, 21205
Not Yet Recruiting
4
Clinical Trial Site
Kansas City, Missouri, United States, 64108
Not Yet Recruiting
5
Rare Disease Research
Hillsborough, North Carolina, United States, 27278
Actively Recruiting
6
Akron Children's Hospital
Akron, Ohio, United States, 44308
Actively Recruiting
7
Clinical Trial Site
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
8
UT Health Austin
Austin, Texas, United States, 78723
Actively Recruiting
9
Carum Research Inc.
Dallas, Texas, United States, 75243
Actively Recruiting
10
Clinical Trial Site
Pilar, Buenos Aires, Argentina
Not Yet Recruiting
11
Clinical Trial Site
Curitiba, Paraná, Brazil
Not Yet Recruiting
12
Clinical Trial Site
Santa Cecília, Porto Alegre, Brazil
Not Yet Recruiting
13
Clinical Trial Site
Marseille, France
Not Yet Recruiting
14
Clinical Trial Site
Paris, France
Not Yet Recruiting
15
Clinical Trial Site
Ramat Gan, Israel
Not Yet Recruiting
16
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Italy
Actively Recruiting
17
Fondazione IRCCS Istituto Neurologico C. Besta
Milan, Italy
Actively Recruiting
18
Clinical Trial Site
Rome, Italy
Not Yet Recruiting
19
Hospital de Santa Maria
Lisbon, Portugal
Actively Recruiting
20
Hospital Santa Joao
Porto, Portugal
Actively Recruiting
21
Clinical Trial Site
London, United Kingdom
Not Yet Recruiting
22
Clinical Trial Site
Oxford, United Kingdom
Not Yet Recruiting
Research Team
P
Patients Contact Trial Recruitment
CONTACT
H
HCPs Contact: Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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