Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Официальное название

GSDIa Disease Monitoring Program

Обзор

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.

Подробное описание

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.

Вмешательства

• Другое No Intervention
  No investigational/study product will be administered in this DMP.

Первичные конечные точки

• Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator [Срок оценки: 10 Years]
• Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies [Срок оценки: 10 Years]
• Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner [Срок оценки: 10 Years]
• Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner [Срок оценки: 10 Years]
• Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only) [Срок оценки: 1 Year]
• Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only) [Срок оценки: 1 Year]

Вторичные конечные точки (12)

• Percent Change Over Time from Baseline in Total Cornstarch Intake [Срок оценки: Baseline, Up to 10 Years]
• Change from Baseline Over Time in Frequency of Cornstarch Intake [Срок оценки: Baseline, Up to 10 Years]
• Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch [Срок оценки: Baseline, Up to 10 Years]
• Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery [Срок оценки: Baseline, Up to 10 Years]
• Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch) [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: Baseline, Up to 10 Years]
• Change from Baseline Over Time in Real-World Overnight Fasting Tolerance [Срок оценки: Baseline, Up to 10 Years]
• Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height [Срок оценки: 10 Years]
• Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight [Срок оценки: 10 Years]
• Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI) [Срок оценки: 10 Years]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 9 центров

• Children's Hospital of Orange County — Orange
• Children's Hospital Colorado — Denver
• University of Connecticut Health Center — Hartford
• University of Michigan — Ann Arbor
• Duke University Medical Center — Durham
• The Cleveland Clinic Foundation — Cleveland
• Children's Hospital of Philadelphia — Philadelphia
• University of Texas Health Science Center at Houston — Houston
• … и ещё 1 центр

Италия · 2 центра

• University of Naples — Naples
• Istituto G Gaslini Ospedale Pediatrico IRCCS — Genoa

Япония · 2 центра

• Osaka City General Hospital — Osaka
• Fujita Health University Hospital — Toyoake

Бразилия · 1 центр

• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre

Канада · 1 центр

• McGill University Health Center — Montreal

Дания · 1 центр

• Rigshospitalet — Copenhagen

Германия · 1 центр

• Universitätsklinikum Hamburg Eppendorf — Hamburg

Нидерланды · 1 центр

• Groningen University — Groningen

Испания · 1 центр

• Hospital Clínico Universitario de Santiago — Santiago

Идентификаторы

NCT: NCT06636383 · DTX401-CL401 · 2023-510219-20-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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