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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GSDIa Disease Monitoring Program
- ClinicalTrials.gov ID
- NCT06636383
- Acronym / sponsor ID
- DTX401-CL401
- Conditions
- Glycogen Storage Disease Type Ia
- Keywords
- Glycogen storage disorder Ia, AAV, Gene therapy, Von Gierke disease, Glucose metabolism disorder, GSD1, GSDIa
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 4 Nov 2024 (actual)
- Primary completion
- Dec 2036 (estimated)
- Study completion
- Dec 2036 (estimated)
- First posted
- 10 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1: DTX401 in Prior Clinical Study | – | Other: No InterventionPatients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401 |
| Group 2: DTX401 in Post-Marketing Setting | – | Other: No InterventionPatients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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.
Key inclusion criteria (4 listed)
- Patient who had:
- 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.
Key exclusion criteria (1 listed)
- Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionNo investigational/study product will be administered in this DMP. | Other | Group 1: DTX401 in Prior Clinical Study, Group 2: DTX401 in Post-Marketing Setting |
Primary outcome
Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator
Time frame: 10 Years
Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies
Time frame: 10 Years
Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
Time frame: 10 Years
Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner
Time frame: 10 Years
Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)
Time frame: 1 Year
Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)
Time frame: 1 Year
22 secondary outcomes
Percent Change Over Time from Baseline in Total Cornstarch Intake
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Frequency of Cornstarch Intake
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch)
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Real-World Overnight Fasting Tolerance
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height
Time frame: 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight
Time frame: 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI)
Time frame: 10 Years
Number of Participants with Clinically Significant Laboratory Values
Time frame: 10 Years
Frequency of Major Clinical Events (MCEs)
Time frame: 10 Years
Frequency of MCEs from Review of Medical Records
Time frame: 10 Years
Change from Baseline in Patient Experience Clinical Interview
Time frame: Baseline, Up to 10 Years
Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score
Time frame: Baseline, Up to 1 Year
Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score
Time frame: 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
Time frame: Baseline, Up to 10 Years
Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months
Time frame: Baseline, Up to 10 Years
Change from Baseline in Work/School Productivity Rating
Time frame: Baseline, Up to 10 Years
Change from Baseline in Employment
Time frame: Baseline, Up to 10 Years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Our Clinical Trial Transparency Commitment-UltragenyxLink provided by the sponsor.
- Ultragenyx Patient Advocacy WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Ultragenyx Pharmaceuticals Inc.
19 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type Ia, run by Ultragenyx Pharmaceutical Inc at 19 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Italy, Japan, Brazil, Canada and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patients Contact: Trial Recruitment (1-888-756-8657, trialrecruitment@ultragenyx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator (time frame: 10 Years). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.