Long-term Extension of GTX-102 in Angelman Syndrome

Part of paid clinical trials in Los Angeles, California.

Sponsor
Ultragenyx Pharmaceutical Inc
Study ID
NCT06415344
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • Angelman Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • GTX-102 — DRUG
    Antisense Oligonucleotide

Study Details

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Key Dates

Start date
Jul 31, 2024
Status verified
May 2026
Primary completion
Feb 28, 2029
Completion
Feb 28, 2029

Study Design

Enrollment
255 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: GTX-102
    Participants will receive GTX-102 via intrathecal lumbar puncture (IT LP) on a intrapatient flexible dosing schedule.

Primary Outcome Measure

Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 5 Years ]

Locations (6)

FacilityCityStateZIPSite coordinators
University of California, Los Angeles (UCLA)Los AngelesCalifornia90095-
University of California, San Diego - Rady Children's HospitalSan DiegoCalifornia92123-
Rare Disease Research, LLCAtlantaGeorgia30329-
Rush University Medical CenterChicagoIllinois60612-
Boston Children's HospitalBostonMassachusetts02115-
Weill Cornell MedicineNew YorkNew York10021-

Find similar trials in Los Angeles, CA

By research site
University of California, Los Angeles (UCLA)· Los Angeles, CAUniversity of California, San Diego - Rady Children's Hospital· San Diego, CARare Disease Research, LLC· Atlanta, GARush University Medical Center· Chicago, ILBoston Children's Hospital· Boston, MAWeill Cornell Medicine· New York, NY

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