An Phase 3 clinical study comparing a vector transferring the gene for Glucose-6-Phosphatase with placebo in adults with Glycogen Storage Disease Type Ia.

You may be able to join if

• "1. Male and female patients ≥ 8 years of age at time of informed consent or assent. 2. Subject has a diagnosis of GSDIa confirmed by deficient enzymatic activity (on liver biopsy), or by molecular testing of G6PC gene revealing 2 pathogenic mutations. In the case where only a single pathogenic mutation is identified, clinical diagnosis is compatible with GSDIa and absence of characteristic features of GSDIb (ie, chronic neutropenia, inflammatory bowel disease). 3. Subject is currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations (Appendix 1) with stable nutrition, glycemic, and clinical status as evidenced by: a. no more than a 10% variation in weekly average daily cornstarch (or equivalent) intake over the last 4 weeks. b. no more than a 25% variation in weekly average daily non-cornstarch carbohydrate over the last 4 weeks. c. No more than 15% variation in weekly percentage of values in the target blood glucose range (60-120 mg/dL) over the last 4 weeks as measured by CGM and corroborated by SMBG. If adequate corroboration is not observed, this assessment should be made by SMBG. d. No hospitalization for hypoglycemia and no severe hypoglycemic event (SHE) during the 4-week period preceding randomization and dosing (see Section 10.4.2 for more detail on SHE), notwithstanding events of hypoglycemia due to unavoidable and unforeseeable events (eg, infection, trauma) that transiently prevent the subject from tolerating enteral intake or acutely change the subject's metabolic demands, provided that the subject quickly returns to their prior physiologic state. 4. Subject is willing 4. Subject is willing and able to comply with study procedures, requirements, and study medication, including periodic inpatient hospitalization or admission in a research facility; CFC studies; frequent blood collection; wearing a CGM device for the duration of the study (and excluding the use of any non-study CGM or flash glucose device); performing capillary glucose measurements according to the protocol using a study approved glucometer (and excluding the use of any other glucometer); completing an eDiary to track daily cornstarch, diet intake, and reasons for doing SMBG routinely throughout the study as required by the protocol; and completing patient-reported questionnaires. Subject must strictly comply with prednisolone/placebo prednisolone prescription including changes in prescription that may be implemented during the study by the Investigator, if needed. (See Section 9.2, Prednisolone Taper.) If < 18 years (or as required by region), has a parent or legal guardian willing and able to assist with study requirements. 5. From the period following informed consent through the duration of participation in the study, female subjects of childbearing potential and fertile male subjects must consent to use highly effective contraception as defined by the Food and Drug Administration (FDA) and Clinical Trial Facilitation Group Recommendations Related to Contraception and Pregnancy Testing in Clinical Trials (Version 1.1 dated 21 Sep 2020). Female subjects must agree not to become pregnant and male subjects must agree not father a child or donate sperm for at least 48 weeks after the last dose of IP if they decide to withdraw early from the study. 6. Subject is willing and able to provide written informed consent after the study has been explained and before any study-related procedures are performed. If < 18 years (or as required by region), willing and able to provide written assent and have a parent or legal guardian willing and able to provide written informed consent after the study has been explained and before any study-related procedures are performed."