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.
Study Type
OBSERVATIONAL
Enrollment
140
No investigational/study product will be administered in this DMP.
Children's Hospital of Orange County
Orange, California, United States
RECRUITINGIncidence 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
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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Children's Hospital Colorado
Denver, Colorado, United States
RECRUITINGUniversity of Connecticut Health Center
Hartford, Connecticut, United States
RECRUITINGUniversity of Michigan
Ann Arbor, Michigan, United States
RECRUITINGDuke University Medical Center
Durham, North Carolina, United States
RECRUITINGThe Cleveland Clinic Foundation
Cleveland, Ohio, United States
NOT_YET_RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGUniversity of Texas Health Science Center at Houston
Houston, Texas, United States
RECRUITINGPrimary Children's Hospital
Salt Lake City, Utah, United States
NOT_YET_RECRUITINGHospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
NOT_YET_RECRUITING...and 9 more locations
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
The following ranges will be assessed: \< 54 mg/dL, \< 60 mg/dL, \< 70 mg/dL, 70-120 mg/dL, \> 120 mg/dL
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
The following ranges will be assessed: \< 54 mg/dL, \< 70 mg/dL
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
The following ranges will be assessed: \< 54 mg/dL, \< 70 mg/dL
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