Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
- Conditions
- Glycogen Storage Disease Type Ia
- Interventions
- Other: No Intervention
- Registration Number
- NCT06636383
- Lead Sponsor
- Ultragenyx Pharmaceutical Inc
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 140
-
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.
- Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Group 1: DTX401 in Prior Clinical Study No Intervention Patients administered DTX401 (full or partial dose) in prior clinical study involving DTX401 Group 2: DTX401 in Post-Marketing Setting No Intervention Patients administered prescribed DTX401(full or partial dose) in a post-marketing setting
- Primary Outcome Measures
Name Time Method Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner 10 Years Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner 10 Years Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies 10 Years Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator 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 Outcome Measures
Name Time Method 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 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
Trial Locations
- Locations (19)
Universitätsklinikum Hamburg Eppendorf
🇩🇪Hamburg, Germany
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Children's Hospital Colorado
🇺🇸Denver, Colorado, United States
University of Connecticut Health Center
🇺🇸Hartford, Connecticut, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
The Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Texas Health Science Center at Houston
🇺🇸Houston, Texas, United States
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
🇧🇷Porto Alegre, Rio Grande do Sul, Brazil
McGill University Health Center
🇨🇦Montréal, Quebec, Canada
Rigshospitalet
🇩🇰Copenhagen, Denmark
University of Naples
🇮🇹Napoli, Campania, Italy
Istituto G Gaslini Ospedale Pediatrico IRCCS
🇮🇹Genoa, Italy
Osaka City General Hospital
🇯🇵Osaka, Japan
Fujita Health University Hospital
🇯🇵Toyoake, Japan
Groningen University
🇳🇱Groningen, Netherlands
Hospital ClÃnico Universitario de Santiago
🇪🇸Santiago, Spain