A clinical study to learn about the effects of a virus that transfers the gene for Glucose- 6-Phosphatase (G6Pase) in adults with Glycogen Storage Disease Type Ia
- Conditions
- Glycogen Storage Disease Type Ia (GSDIa).MedDRA version: 20.1Level: LLTClassification code 10056911Term: Glycogen storage disease type IASystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2018-004473-27-NL
- Lead Sponsor
- ltragenyx Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 12
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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 11
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
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
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine the long-term safety of DTX401 following a single IV dose in adults with GSDIa.;Secondary Objective: To evaluate the long-term effect of DTX401 on symptom-free euglycemia in a setting of a controlled fasting challenge.;Primary end point(s): The incidence of AEs and SAEs for each dose level assessed by severity and relationship to IP.;Timepoint(s) of evaluation of this end point: From the time the subject signs the Informed Consent Form through the end of the study/early withdrawal visit.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The change from Day 0 (Study 401GSDIA01) in time to first hypoglycemic event during a controlled fasting challenge over time to 260 weeks by dose level, following IV administration of DTX401.<br>;Timepoint(s) of evaluation of this end point: Weeks 52, 78, 104, 130, 156, 182, 208, 234 and 260.