Long-Term Follow-up 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)

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Brief Title

Long-Term Follow-up 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)

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)

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.
    


Study Type

Observational


Primary Outcome

Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs

Secondary Outcome

 Change in Time to First Hypoglycemic Event During a Controlled Fasting Challenge from Day 0 (Study 401GSDIA01) to 260 weeks

Condition

Glycogen Storage Disease Type IA

Intervention

No intervention

Study Arms / Comparison Groups

 All Participants
Description:  All participants enrolled in study 401GSDIA02 will have received a single IV dose of DTX401 during their participation in study 401GSDIA01 (NCT03517085).

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

12

Start Date

July 15, 2019

Completion Date

December 2025

Primary Completion Date

December 2025

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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Director, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT03970278

Organization ID

401GSDIA02

Secondary IDs

2018-004473-27

Responsible Party

Sponsor

Study Sponsor

Ultragenyx Pharmaceutical Inc


Study Sponsor

Medical Director, Study Director, Ultragenyx Pharmaceutical Inc


Verification Date

July 2022