A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome
Ultragenyx Pharmaceutical Inc
Summary
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
Description
This basket study is designed to evaluate safety and efficacy of GTX-102 in participants with Angelman syndrome across genotypes and age groups. The study consists of subprotocols A, B, C and D. All subprotocols are open-label and follow the same design which includes a Screening, Loading and Maintenance period. Subprotocols A, B and C are single arm only. In subprotocol D, participants are randomized 2:1 to a GTX-102 group or a No Treatment group. The No Treatment group follows the same schedule of events as all other groups after completion of the No Treatment period. Participants from all t…
Eligibility
- Age range
- 1–64 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Signed informed consent from parent(s) or legal guardian(s) 2. Males and females of the following ages and genotypes at time of informed consent: 1. Subprotocol A: ≥ 1 to \< 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman syndrome 2. Subprotocol B: ≥ 4 to \< 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman syndrome 3. Subprotocol C: ≥ 18 to \< 65 years of age with a genetically confirmed diagnosis of Angelman syndrome, any genotype 4. Subprotocol D: ≥ 4 to \< 18 years of age with a genetically confir…
Interventions
- OtherNo intervention
During the no treatment period participants do not receive any study drug
- DrugGTX-102
antisense oligonucleotide
Locations (22)
- Cedars Sinai Medical CenterLos Angeles, California
- Rush University Medical CenterChicago, Illinois
- Clinical Trial SiteBaltimore, Maryland
- Clinical Trial SiteKansas City, Missouri
- Rare Disease ResearchHillsborough, North Carolina
- Akron Children's HospitalAkron, Ohio