Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Ultragenyx

Status: Completed

Conditions: Von Gierke's Disease (GSD Type Ia); Glycogen Storage Disease Type IA

Treatments: Other: No intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT03970278

2023-504004-29-00 (EU Trial (CTIS) Number)

401GSDIA02

2018-004473-27 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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. Participants will be followed in study 401GSDIA02 for at least 4 years, and up to 6 years after administration of DTX401.

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

12 participants in 4 patient groups

DTX401 Cohort 1

Description:

Participants received a single intravenous (IV) dose of 2.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with a reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

Treatment:

Other: No intervention

DTX401 Cohort 2

Description:

Participants received a single IV dose of 6.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with a reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

Treatment:

Other: No intervention

DTX401 Cohort 3

Description:

Participants received a single IV dose of 6.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with an optimized reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

Treatment:

Other: No intervention

DTX401 Cohort 4

Description:

Participants received a single IV dose of 6.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with a prophylactic steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

Treatment:

Other: No intervention