Print Send My Information

Purpose

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.

Condition

Eligibility

Eligible Ages
Over 2 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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-marketing setting (Group 2) - Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.

Exclusion Criteria

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

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Group 1: DTX401 in Prior Clinical Study Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
  • Other: No Intervention
    No investigational/study product will be administered in this DMP.
Group 2: DTX401 in Post-Marketing Setting Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting
  • Other: No Intervention
    No investigational/study product will be administered in this DMP.

Recruiting Locations

Children's Hospital of Orange County
Orange 5379513, California 5332921 92868

Children's Hospital Colorado
Denver 5419384, Colorado 5417618 80045

University of Connecticut Health Center
Hartford 4835797, Connecticut 4831725 06106

University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109

Duke University Medical Center
Durham 4464368, North Carolina 4482348 27710

Children's Hospital of Philadelphia
Philadelphia 4560349, Pennsylvania 6254927 19104

University of Texas Health Science Center at Houston
Houston 4699066, Texas 4736286 77030

More Details

NCT ID
NCT06636383
Status
Recruiting
Sponsor
Ultragenyx Pharmaceutical Inc

Study Contact

Patients Contact: Trial Recruitment
1-888-756-8657
trialrecruitment@ultragenyx.com

Detailed 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.

For help using this page: trialstoday@researchmatch.org or call 855-514-7001

ResearchMatch is funded in part by the National Institutes of Health (NIH) Clinical and Translational Science Award (CTSA) program, led by the NIH’s National Center for Advancing Translational Sciences (NCATS), grants UL1TR000445 and 1U54RR032646-01, and grant U24TR001579 from NCATS and the National Library of Medicine. The content of this website is solely the responsibility of ResearchMatch and Vanderbilt University and does not necessarily represent the official views of the NIH, NCATS, or NLM.

Vanderbilt University Medical Center