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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03970278
Other study ID # 401GSDIA02
Secondary ID 2018-004473-27
Status Active, not recruiting
Phase
First received
Last updated
Start date July 15, 2019
Est. completion date December 2026

Study information

Verified date May 2024
Source Ultragenyx Pharmaceutical Inc
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 12
Est. completion date December 2026
Est. primary completion date December 2026
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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.

Study Design


Intervention

Other:
No intervention
No intervention

Locations

Country Name City State
Canada Montreal Children Hospital, McGill University Health Centre Montréal Quebec
Netherlands University Medical Center Groningen Groningen
Spain Complejo Hospitalario Universitario de Santiago Santiago De Compostela A Coruna
United States Michigan Medicine University of Michigan Ann Arbor Michigan
United States UCONN Health Farmington Connecticut
United States University of Texas Health Science Center at Houston Houston Texas

Sponsors (1)

Lead Sponsor Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Canada,  Netherlands,  Spain, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs Up to 312 weeks following DTX401 administration
Secondary Change in Time to First Hypoglycemic Event During a Controlled Fasting Challenge from Day 0 (Study 401GSDIA01) to 312 weeks Up to 312 weeks following DTX401 administration
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