Monitoring program for patients with Glycogen Storage Disease Type Ia

GSDIa Disease Monitoring Program

This study is tracking the long-term health and safety of a treatment called DTX401 in people with Glycogen Storage Disease Type Ia to see how it helps them over at least 10 years.

Quick facts

• Study type: Observational
• Enrollment: 140 (estimated)
• Ages: 2 Years and up
• Sex: All
• Sponsor: Ultragenyx Pharmaceutical Inc (industry)
• Locations: 19 sites (Orange, California and 18 other locations)
• Trial ID: NCT06636383 on ClinicalTrials.gov

What this trial studies

This observational program aims to evaluate the long-term safety and effectiveness of DTX401 in patients with Glycogen Storage Disease Type Ia over a period of at least 10 years. Participants who have received DTX401 in previous studies or post-marketing settings will be followed to gather data on their health outcomes. The study is multicenter and prospective, focusing on the ongoing health and metabolic status of these patients without any new interventions being administered.

Who should consider this trial

Good fit: Ideal candidates are patients who have previously received DTX401, either in a clinical study or post-marketing setting.

Not a fit: Patients who have not received DTX401 or have conditions that interfere with study participation may not benefit from this program.

Why it matters

Potential benefit: If successful, this program could provide valuable insights into the long-term effects of DTX401, potentially improving management strategies for patients with GSDIa.

How similar studies have performed: While this program is observational, similar long-term monitoring studies have shown success in providing critical data on treatment outcomes in rare diseases.

Eligibility criteria

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

Where this trial is running

Orange, California and 18 other locations

• Children's Hospital of Orange County — Orange, California, United States (RECRUITING)
• Children's Hospital Colorado — Denver, Colorado, United States (RECRUITING)
• University of Connecticut Health Center — Hartford, Connecticut, United States (RECRUITING)
• University of Michigan — Ann Arbor, Michigan, United States (RECRUITING)
• Duke University Medical Center — Durham, North Carolina, United States (RECRUITING)
• The Cleveland Clinic Foundation — Cleveland, Ohio, United States (NOT_YET_RECRUITING)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (RECRUITING)
• University of Texas Health Science Center at Houston — Houston, Texas, United States (RECRUITING)
• Primary Children's Hospital — Salt Lake City, Utah, United States (NOT_YET_RECRUITING)
• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Rio Grande do Sul, Brazil (RECRUITING)
• McGill University Health Center — Montreal, Quebec, Canada (RECRUITING)
• Rigshospitalet — Copenhagen, Denmark (RECRUITING)
• Universitätsklinikum Hamburg Eppendorf — Hamburg, Germany (RECRUITING)
• University of Naples — Naples, Campania, Italy (RECRUITING)
• Istituto G Gaslini Ospedale Pediatrico IRCCS — Genoa, Italy (RECRUITING)
• Osaka City General Hospital — Osaka, Japan (RECRUITING)
• Fujita Health University Hospital — Toyoake, Japan (RECRUITING)
• Groningen University — Groningen, Netherlands (RECRUITING)
• Hospital Clínico Universitario de Santiago — Santiago, Spain (RECRUITING)

Study contacts

• Study coordinator: Patients Contact: Trial Recruitment

Conditions: Glycogen Storage Disease Type Ia, Glycogen storage disorder Ia, AAV, Gene therapy, Von Gierke disease, Glucose metabolism disorder, GSD1, GSDIa