Glycogen Storage Disease Type IaNCT06636383
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
An observational study of Glycogen Storage Disease Type Ia, run by Ultragenyx Pharmaceutical Inc at 19 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Glycogen Storage Disease Type Ia
- How many people
- 140 participants
- Where
- 19 sitesin 9 countries
- Study dates
- 4 Nov 2024to Dec 2036
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All participants who received DTX401 in a qualifying clinical study will be expected and asked to participate in the DMP upon completion or early termination (ET) from the parent study, as required by regulatory agencies for all gene therapy studies.
Who may be able to join
- 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.
Who cannot join
- Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
Trial locations and contacts
19 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
19 sites in 9 countries
United States9 sites, 9 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Not yet recruiting
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
- Recruiting
Children's Hospital Colorado
Denver, Colorado, 80045
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
- Recruiting
University of Connecticut Health Center
Hartford, Connecticut, 06106
- Recruiting
University of Texas Health Science Center at Houston
Houston, Texas, 77030
- Recruiting
Children's Hospital of Orange County
Orange, California, 92868
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Not yet recruiting
Primary Children's Hospital
Salt Lake City, Utah, 84108
Italy2 sites, 2 recruiting
- Recruiting
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa, 16147
- Recruiting
University of Naples
Naples, Campania, 80131
Japan2 sites, 2 recruiting
- Recruiting
Osaka City General Hospital
Osaka, 534-0021
- Recruiting
Fujita Health University Hospital
Toyoake, 470-1192
Brazil1 site, 1 recruiting
- Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, 90035-903
Canada1 site, 1 recruiting
- Recruiting
McGill University Health Center
Montreal, Quebec, H4A3J1
Germany1 site, 1 recruiting
- Recruiting
Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246
Netherlands1 site, 1 recruiting
- Recruiting
Groningen University
Groningen, Provincie Groningen, 9700 RB
Spain1 site, 1 recruiting
- Recruiting
Hospital Clínico Universitario de Santiago
Santiago, 15706
About this study
Official title: GSDIa Disease Monitoring Program
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.
Read the 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.
- Conditions
- Glycogen Storage Disease Type Ia
- Being tested
- No Intervention
- Keywords
- Glycogen storage disorder Ia, AAV, Gene therapy, Von Gierke disease, Glucose metabolism disorder, GSD1, GSDIa
- Medical subject headings
- Hepatorenal form of glycogen storage disease, Glycogen Storage Disease Type I, Glucose Metabolism Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
No investigational/study product will be administered in this DMP.
| Study group | Role | What they receive |
|---|---|---|
| Group 1: DTX401 in Prior Clinical Study | Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401 Receives: Other: No Intervention | |
| Group 2: DTX401 in Post-Marketing Setting | Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator
Measured: 10 Years
Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies
Measured: 10 Years
Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
Measured: 10 Years
Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner
Measured: 10 Years
Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)
Measured: 1 Year
Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)
Measured: 1 Year
Secondary outcomes (22)
Percent Change Over Time from Baseline in Total Cornstarch Intake
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Frequency of Cornstarch Intake
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch)
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Daytime and Nighttime Percentage of Glucose Values During the 2 Weeks Leading Up to the Assessment Visits
The following ranges will be assessed: < 54 mg/dL, < 60 mg/dL, < 70 mg/dL, 70-120 mg/dL, > 120 mg/dL
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Total Number of Hypoglycemic Events on Continuous Glucose Monitoring (CGM) During the 2 Weeks Leading Up to the Assessment Visits
The following ranges will be assessed: < 54 mg/dL, < 70 mg/dL
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Total Duration of Hypoglycemic Events on CGM During the 2 Weeks Leading Up to the Assessment Visits
The following ranges will be assessed: < 54 mg/dL, < 70 mg/dL
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Real-World Overnight Fasting Tolerance
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height
Measured: 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight
Measured: 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI)
Measured: 10 Years
Number of Participants with Clinically Significant Laboratory Values
Measured: 10 Years
Frequency of Major Clinical Events (MCEs)
Measured: 10 Years
Frequency of MCEs from Review of Medical Records
Measured: 10 Years
Change from Baseline in Patient Experience Clinical Interview
Measured: Baseline, Up to 10 Years
Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score
Measured: Baseline, Up to 1 Year
Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score
Measured: Baseline, Up to 1 Year
Change from Baseline Over Time in Annual Number of GSDIa Related Hospitalizations, Emergency Room Visits, Outpatient Visits, and Hospital Days
Measured: Baseline, Up to 10 Years
Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months
Measured: Baseline, Up to 10 Years
Change from Baseline in Work/School Productivity Rating
Measured: Baseline, Up to 10 Years
Change from Baseline in Employment
Measured: Baseline, Up to 10 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2024
- Study start4 Nov 2024
- Primary completionDec 2036 (estimated)
- Study completionDec 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IaStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceuticals Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06636383
- Sponsor's protocol ID
- DTX401-CL401
- Other IDs
- 2023-510219-20-00, 2023
- Status last verified
- Oct 2026
- First submitted
- 20 Sep 2024
- Last updated
- 14 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No