Glycogen Storage Disease Type Ia
Conditions
Keywords
Glycogen storage disorder Ia, AAV, Gene therapy, Von Gierke disease, Glucose metabolism disorder, GSD1, GSDIa
Brief 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.
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.
Interventions
OTHERNo Intervention
No investigational/study product will be administered in this DMP.
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| 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 | Baseline, Up to 10 Years | The following ranges will be assessed: \< 54 mg/dL, \< 60 mg/dL, \< 70 mg/dL, 70-120 mg/dL, \> 120 mg/dL |
| 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 | Baseline, Up to 10 Years | The following ranges will be assessed: \< 54 mg/dL, \< 70 mg/dL |
| Change from Baseline Over Time in Total Duration of Hypoglycemic Events on CGM During the 2 Weeks Leading Up to the Assessment Visits | Baseline, Up to 10 Years | The following ranges will be assessed: \< 54 mg/dL, \< 70 mg/dL |
| 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 | — |
Countries
Brazil, Canada, Denmark, Germany, Italy, Japan, Netherlands, Spain, United States
Contacts
CONTACTPatients Contact: Trial Recruitment
CONTACTHCPs Contact: Medical Information
STUDY_DIRECTORMedical Director
Ultragenyx Pharmaceuticals Inc.
Outcome results
None listed