E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Glycogen Storage Disease Type Ia (GSDIa). |
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E.1.1.1 | Medical condition in easily understood language |
Inherited disorder causing decrease of glucose. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056911 |
E.1.2 | Term | Glycogen storage disease type IA |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the long-term safety of DTX401 following a single IV dose in adults with GSDIa. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the long-term effect of DTX401 on symptom-free euglycemia in a setting of a controlled fasting challenge. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The incidence of AEs and SAEs for each dose level assessed by severity and relationship to IP. |
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E.5.1.1 | 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. |
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E.5.2 | 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.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Weeks 52, 78, 104, 130, 156, 182, 208, 234 and 260. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Long-term follow-up study |
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E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
United States |
Netherlands |
Spain |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 2 |