RECRUITING

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Talk to us

Conditions

Glycogen Storage Disease Type IaGlycogen storage disorder IaAAVGene therapyVon Gierke diseaseGlucose metabolism disorderGSD1GSDIa

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

GSDIa Disease Monitoring Program

Quick Facts

Study Start:2024-11-04
Study Completion:2036-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06636383

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Patient who had:
  2. * DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or
  3. * Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)
  4. * 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.
  1. * Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Contacts and Locations

Study Contact

Patients Contact: Trial Recruitment
CONTACT
1-888-756-8657
trialrecruitment@ultragenyx.com
HCPs Contact: Medical Information
CONTACT
1-888-756-8657
medinfo@ultragenyx.com

Principal Investigator

Medical Director
STUDY_DIRECTOR
Ultragenyx Pharmaceuticals Inc.

Study Locations (Sites)

Children's Hospital of Orange County
Orange, California, 92868
United States
Children's Hospital Colorado
Denver, Colorado, 80045
United States
University of Connecticut Health Center
Hartford, Connecticut, 06106
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
United States
Duke University Medical Center
Durham, North Carolina, 27710
United States
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030
United States
Primary Children's Hospital
Salt Lake City, Utah, 84108
United States

Collaborators and Investigators

Sponsor: Ultragenyx Pharmaceutical Inc

  • Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceuticals Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-11-04
Study Completion Date2036-12

Study Record Updates

Study Start Date2024-11-04
Study Completion Date2036-12

Terms related to this study

Keywords Provided by Researchers

  • Glycogen storage disorder Ia
  • AAV
  • Gene therapy
  • Von Gierke disease
  • Glucose metabolism disorder
  • GSD1
  • GSDIa

Additional Relevant MeSH Terms

  • Glycogen Storage Disease Type Ia