Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Part of paid clinical trials in Orange, California.

Sponsor
Ultragenyx Pharmaceutical Inc
Study ID
NCT06636383
Status
Recruiting
Apply to this trial

Conditions

  • Glycogen Storage Disease Type Ia

Eligibility Criteria

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

Interventions

  • No Intervention — OTHER
    No investigational/study product will be administered in this DMP.

Study Details

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

Key Dates

Start date
Nov 4, 2024
Status verified
May 2026
Primary completion
Dec 31, 2036
Completion
Dec 31, 2036

Study Design

Enrollment
140 participants (estimated)

Arms

  • Arm: Group 1: DTX401 in Prior Clinical Study
    Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
  • Arm: Group 2: DTX401 in Post-Marketing Setting
    Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting

Primary Outcome Measure

Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator [ Time Frame: 10 Years ]

Central Contacts

Locations (9)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange CountyOrangeCalifornia92868-
Children's Hospital ColoradoDenverColorado80045-
University of Connecticut Health CenterHartfordConnecticut06106-
University of MichiganAnn ArborMichigan48109-
Duke University Medical CenterDurhamNorth Carolina27710-
The Cleveland Clinic FoundationClevelandOhio44195-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
University of Texas Health Science Center at HoustonHoustonTexas77030-
Primary Children's HospitalSalt Lake CityUtah84108-

Find similar trials in Orange, CA

By research site
Children's Hospital of Orange County· Orange, CAChildren's Hospital Colorado· Denver, COUniversity of Connecticut Health Center· Hartford, CTUniversity of Michigan· Ann Arbor, MIDuke University Medical Center· Durham, NCThe Cleveland Clinic Foundation· Cleveland, OH

Related Studies