Clinical trial NCT06636383: Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Official title: GSDIa Disease Monitoring Program
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Glycogen Storage Disease Type Ia
Keywords: Glycogen storage disorder Ia
AAV
Gene therapy
Von Gierke disease
Glucose metabolism disorder
GSD1
GSDIa
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2024-10-10
Enrollment: 140
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator
timeFrame: 10 Years
measure: Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies
timeFrame: 10 Years
measure: Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
timeFrame: 10 Years
measure: Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner
timeFrame: 10 Years
measure: Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)
timeFrame: 1 Year
measure: Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)
timeFrame: 1 Year
Secondary outcomes:
measure: Percent Change Over Time from Baseline in Total Cornstarch Intake
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Frequency of Cornstarch Intake
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch)
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Daytime and Nighttime Percentage of Glucose Values During the 2 Weeks Leading Up to the Assessment Visits
timeFrame: Baseline, Up to 10 Years
measure: 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
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Total Duration of Hypoglycemic Events on CGM During the 2 Weeks Leading Up to the Assessment Visits
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Real-World Overnight Fasting Tolerance
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height
timeFrame: 10 Years
measure: Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight
timeFrame: 10 Years
measure: Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI)
timeFrame: 10 Years
measure: Number of Participants with Clinically Significant Laboratory Values
timeFrame: 10 Years
measure: Frequency of Major Clinical Events (MCEs)
timeFrame: 10 Years
measure: Frequency of MCEs from Review of Medical Records
timeFrame: 10 Years
measure: Change from Baseline in Patient Experience Clinical Interview
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score
timeFrame: Baseline, Up to 1 Year
measure: Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score
timeFrame: Baseline, Up to 1 Year
measure: Change from Baseline Over Time in Annual Number of GSDIa Related Hospitalizations, Emergency Room Visits, Outpatient Visits, and Hospital Days
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline in Work/School Productivity Rating
timeFrame: Baseline, Up to 10 Years
measure: Change from Baseline in Employment
timeFrame: Baseline, Up to 10 Years
Other outcomes:
Locations:
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Denver
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Hartford
country: United States
facility: University of Connecticut Health Center
state: Connecticut
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
state: Rio Grande do Sul
city: Montréal
country: Canada
facility: McGill University Health Center
state: Quebec
city: Copenhagen
country: Denmark
facility: Rigshospitalet
city: Hamburg
country: Germany
facility: Universitätsklinikum Hamburg Eppendorf
city: Napoli
country: Italy
facility: University of Naples
state: Campania
city: Genoa
country: Italy
facility: Istituto G Gaslini Ospedale Pediatrico IRCCS
city: Osaka
country: Japan
facility: Osaka City General Hospital
city: Toyoake
country: Japan
facility: Fujita Health University Hospital
city: Groningen
country: Netherlands
facility: Groningen University
city: Santiago
country: Spain
facility: Hospital Clínico Universitario de Santiago
Study officials:
affiliation: Ultragenyx Pharmaceuticals Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: