Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Last updated: May 6, 2025
Sponsor: Ultragenyx Pharmaceutical Inc
Overall Status: Active - Recruiting

Phase

N/A

Condition

Liver Disease

Liver Disorders

Treatment

No Intervention

Clinical Study ID

NCT06636383
DTX401-CL401
2023-510219-20-00
  • Ages > 2
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patient who had:

  • DTX401 (full or partial dose) administered in a parent clinical study (Group 1)or

  • Prescribed DTX401(full or partial dose) administered in a post-marketingsetting (Group 2)

  • Patient is willing and able to provide informed consent after the nature of thestudy has been explained, and prior to any research-related assessments orprocedures. If a minor or an adult with cognitive limitations, the patient iswilling and able (if possible) to provide assent and have a legally authorizedrepresentative provide informed consent after the nature of the study has beenexplained, and prior to any research-related assessments or procedures.

Exclusion

Exclusion Criteria:

  • Presence of any condition that would interfere with study participation,interpretation of results or affect patient's safety in the opinion of theInvestigator

Study Design

Total Participants: 140
Treatment Group(s): 1
Primary Treatment: No Intervention
Phase:
Study Start date:
November 04, 2024
Estimated Completion Date:
December 31, 2036

Study Description

The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.

Connect with a study center

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    Porto Alegre, Rio Grande do Sul 90035-903
    Brazil

    Site Not Available

  • McGill University Health Center

    Montréal, Quebec H4A3J1
    Canada

    Active - Recruiting

  • Rigshospitalet

    Copenhagen, 2700
    Denmark

    Site Not Available

  • Universitätsklinikum Hamburg Eppendorf

    Hamburg, 20246
    Germany

    Site Not Available

  • University of Naples

    Napoli, Campania 80131
    Italy

    Site Not Available

  • Istituto G Gaslini Ospedale Pediatrico IRCCS

    Genoa, 16147
    Italy

    Site Not Available

  • Osaka City General Hospital

    Osaka, 534-0021
    Japan

    Site Not Available

  • Fujita Health University Hospital

    Toyoake, 470-1192
    Japan

    Site Not Available

  • Groningen University

    Groningen, 9700 RB
    Netherlands

    Active - Recruiting

  • Hospital Clínico Universitario de Santiago

    Santiago, 15706
    Spain

    Active - Recruiting

  • Children's Hospital of Orange County

    Orange, California 92868
    United States

    Site Not Available

  • Children's Hospital Colorado

    Denver, Colorado 80045
    United States

    Site Not Available

  • University of Connecticut Health Center

    Hartford, Connecticut 06106
    United States

    Active - Recruiting

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • Icahn School of Medicine at Mount Sinai

    New York, New York 10029
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27710
    United States

    Site Not Available

  • The Cleveland Clinic Foundation

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Texas Health Science Center at Houston

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Primary Children's Hospital

    Salt Lake City, Utah 84108
    United States

    Site Not Available

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