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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-510219-20-00 | EU Trial (CTIS) Number |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group 1: DTX401 in Prior Clinical Study | Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401 |
| |
| Group 2: DTX401 in Post-Marketing Setting | Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | No investigational/study product will be administered in this DMP. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator | 10 Years | |
| Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies | 10 Years | |
| Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner | 10 Years | |
| Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner | 10 Years | |
| Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only) | 1 Year | |
| Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only) | 1 Year |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change Over Time from Baseline in Total Cornstarch Intake | Baseline, Up to 10 Years | |
| Change from Baseline Over Time in Frequency of Cornstarch Intake | Baseline, Up to 10 Years | |
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Inclusion Criteria:
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.
Exclusion Criteria:
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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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Patients Contact: Trial Recruitment | Contact | 1-888-756-8657 | trialrecruitment@ultragenyx.com | |
| HCPs Contact: Medical Information | Contact | 1-888-756-8657 | medinfo@ultragenyx.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceuticals Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Orange County | Recruiting | Orange | California | 92868 | United States | |
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| Label | URL |
|---|---|
| Our Clinical Trial Transparency Commitment-Ultragenyx | View source |
| Ultragenyx Patient Advocacy Website | View source |
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| Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch |
| Baseline, Up to 10 Years |
| Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery | Baseline, Up to 10 Years |
| Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch) | 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 | 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 | 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 | Baseline, Up to 10 Years |
| Change from Baseline Over Time in Real-World Overnight Fasting Tolerance | Baseline, Up to 10 Years |
| Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height | 10 Years |
| Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight | 10 Years |
| Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI) | 10 Years |
| Number of Participants with Clinically Significant Laboratory Values | 10 Years |
| Frequency of Major Clinical Events (MCEs) | 10 Years |
| Frequency of MCEs from Review of Medical Records | 10 Years |
| Change from Baseline in Patient Experience Clinical Interview | Baseline, Up to 10 Years |
| Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score | Baseline, Up to 1 Year |
| Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score | 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 | Baseline, Up to 10 Years |
| Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months | Baseline, Up to 10 Years |
| Change from Baseline in Work/School Productivity Rating | Baseline, Up to 10 Years |
| Change from Baseline in Employment | Baseline, Up to 10 Years |
| Children's Hospital Colorado |
| Recruiting |
| Denver |
| Colorado |
| 80045 |
| United States |
| University of Connecticut Health Center | Recruiting | Hartford | Connecticut | 06106 | United States |
| University of Michigan | Recruiting | Ann Arbor | Michigan | 48109 | United States |
| Duke University Medical Center | Recruiting | Durham | North Carolina | 27710 | United States |
| The Cleveland Clinic Foundation | Not yet recruiting | Cleveland | Ohio | 44195 | United States |
| Children's Hospital of Philadelphia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| University of Texas Health Science Center at Houston | Recruiting | Houston | Texas | 77030 | United States |
| Primary Children's Hospital | Not yet recruiting | Salt Lake City | Utah | 84108 | United States |
| Hospital de Clinicas de Porto Alegre (HCPA) - PPDS | Recruiting | Porto Alegre | Rio Grande do Sul | 90035-903 | Brazil |
| McGill University Health Center | Recruiting | Montreal | Quebec | H4A3J1 | Canada |
| Rigshospitalet | Recruiting | Copenhagen | 2700 | Denmark |
| Universitätsklinikum Hamburg Eppendorf | Recruiting | Hamburg | 20246 | Germany |
| University of Naples | Recruiting | Naples | Campania | 80131 | Italy |
| Istituto G Gaslini Ospedale Pediatrico IRCCS | Recruiting | Genoa | 16147 | Italy |
| Osaka City General Hospital | Recruiting | Osaka | 534-0021 | Japan |
| Fujita Health University Hospital | Recruiting | Toyoake | 470-1192 | Japan |
| Groningen University | Recruiting | Groningen | Provincie Groningen | 9700 RB | Netherlands |
| Hospital ClĂnico Universitario de Santiago | Recruiting | Santiago | 15706 | Spain |
| ID | Term |
|---|---|
| C538655 | Hepatorenal form of glycogen storage disease |
| D005953 | Glycogen Storage Disease Type I |
| D044882 | Glucose Metabolism Disorders |
| ID | Term |
|---|---|
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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