A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Part of paid clinical trials in Los Angeles, California.

Sponsor
Ultragenyx Pharmaceutical Inc
Study ID
NCT07157254
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Angelman Syndrome

Eligibility Criteria

Sex
ALL
Age
1 Year - 64 Years
Healthy Volunteers
Not accepted

Interventions

  • No intervention — OTHER
    During the no treatment period participants do not receive any study drug
  • GTX-102 — DRUG
    antisense oligonucleotide

Study Details

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Key Dates

Start date
Oct 13, 2025
Status verified
May 2026
Primary completion
Jan 31, 2030
Completion
Jan 31, 2030

Study Design

Enrollment
60 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Subprotocol A GTX-102
    Participants with deletion-type Angelman syndrome, ≥1 to \<4 years of age will receive increasing doses of GTX-102 via intrathecal (IT) injection until the target dose is achieved. Dosing occurs every 3 months (Q3M) thereafter.
  • Experimental: Subprotocol B GTX-102
    Participants with paternal uniparental disomy (UPD)/imprinting center defect (ICD) Angelman syndrome, ≥4 to \<18 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.
  • Experimental: Subprotocol C GTX-102
    Participants with all genotypes of Angelman syndrome, ≥18 to \<65 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.
  • Experimental: Subprotocol D GTX-102
    Participants with mutation-type Angelman syndrome, ≥4 to \<18 years of age will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.
  • Experimental: Subprotocol D No Intervention then GTX-102
    Participants with mutation-type Angelman syndrome, ≥4 to \<18 years of age will receive no treatment during the initial period. At the end of the no treatment period, participants will receive increasing doses of GTX-102 via IT injection until the target dose is achieved. Dosing occurs Q3M thereafter.

Primary Outcome Measure

Subprotocol A/B/C/D: Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), Severe Events, and Events Related to Investigational Product, Procedure, and Premedication [ Time Frame: Up to Day 506 ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
Cedars Sinai Medical CenterLos AngelesCalifornia90048-
Rush University Medical CenterChicagoIllinois60612-
Clinical Trial SiteBaltimoreMaryland21205-
Clinical Trial SiteKansas CityMissouri64108-
Rare Disease ResearchHillsboroughNorth Carolina27278-
Akron Children's HospitalAkronOhio44308-
UT Health AustinAustinTexas78723-
Carum Research Inc.DallasTexas75243-

Find similar trials in Los Angeles, CA

By research site
Cedars Sinai Medical Center· Los Angeles, CARush University Medical Center· Chicago, ILClinical Trial Site· Baltimore, MDClinical Trial Site· Kansas City, MORare Disease Research· Hillsborough, NCAkron Children's Hospital· Akron, OH

Related Studies