Monitoring program for patients with Glycogen Storage Disease Type Ia
Official title: GSDIa Disease Monitoring Program
Run by Ultragenyx Pharmaceutical Inc · NCT06636383 · Data updated 2026-11-12
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 |
|---|---|
| Ages | 2 Years and up |
| Sex | All |
| Enrollment | 140 (estimated) |
| Sponsor | Ultragenyx Pharmaceutical Inc Industry-sponsored |
| Locations | 19 sites (Orange, California and 18 other locations) |
| Status | Recruiting |
| Started | November 4, 2024 |
| Main results expected | December 2036 |
| Study ends | December 2036 |
| Registry record updated | October 14, 2026 |
| Trial ID | NCT06636383 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients who have previously received DTX401, either in a clinical study or post-marketing setting.
✕Probably not a fit
Patients who have not received DTX401 or have conditions that interfere with study participation may not benefit from this program.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
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.
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 gone: While this program is observational, similar long-term monitoring studies have shown success in providing critical data on treatment outcomes in rare diseases.
Who can join: the full rules
Show full inclusion / exclusion 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 it's running
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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 -
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States Recruiting
Show 13 more locations
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University of Texas Health Science Center at Houston
Houston, Texas, United States 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, Provincie Groningen, Netherlands Recruiting -
Hospital Clínico Universitario de Santiago
Santiago, Spain Recruiting -
The Cleveland Clinic Foundation
Cleveland, Ohio, United States Not yet recruiting -
Primary Children's Hospital
Salt Lake City, Utah, United States Not yet recruiting
Study contacts
- Study coordinator: Patients Contact: Trial Recruitment
- Email: trialrecruitment@ultragenyx.com
- Phone: 1-888-756-8657
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Home lactate and Accu‑Chek glucose meter accuracy for people with glycogen storage disease
This project will see if a handheld Lactate Plus meter and an Accu‑Chek glucometer give results that match lab blood tests in people with glycogen storage disease types Ia, Ib, and XI.
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Evaluating the accuracy of home lactate meters in patients with Glycogen Storage Disease Type Ia
This study tests if home lactate meters give accurate readings for people with Glycogen Storage Disease Type Ia by comparing them to lab results.
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Evaluating BEAM-301 for treating Glycogen Storage Disease Type Ia
This study is testing a new treatment called BEAM-301 to see if it is safe and effective for adults with Glycogen Storage Disease Type Ia who have a specific genetic variant.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.