Clinical trial NCT03970278: Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
Official title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Glycogen Storage Disease Type IA
Von Gierke's Disease (GSD Type Ia)
Keywords: AAV
gene therapy
glucose metabolism disorder
carbohydrate metabolism, inborn errors
genetic diseases
Inborn Metabolic Diseases
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2019-05-31
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects 18 years of age or older with GSDIa previously enrolled in 401GSDIA01.

Brief summary: The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

Detailed description: Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02.

Eligibility criteria: Inclusion Criteria:

1. Received DTX401 in study 401GSDIA01.
2. Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed.
3. Willing and able to comply with all scheduled study visits, procedures, and requirements.

Exclusion Criteria:

1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
2. Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs
timeFrame: Up to 312 weeks following DTX401 administration
Secondary outcomes:
measure: Change in Time to First Hypoglycemic Event During a Controlled Fasting Challenge from Day 0 (Study 401GSDIA01) to 312 weeks
timeFrame: Up to 312 weeks following DTX401 administration
Other outcomes:
Locations:
city: Farmington
country: United States
facility: UCONN Health
state: Connecticut
city: Ann Arbor
country: United States
facility: Michigan Medicine University of Michigan
state: Michigan
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
city: Montréal
country: Canada
facility: Montreal Children Hospital, McGill University Health Centre
state: Quebec
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Santiago De Compostela
country: Spain
facility: Complejo Hospitalario Universitario de Santiago
state: A Coruna
Study officials:
affiliation: Ultragenyx Pharmaceutical Inc
name: Medical Director
role: STUDY_DIRECTOR
References: