Glycogen storage disease tyep 1b, neutropenia Glycogen storage disease tyep 1b, neutropenia
Conditions
Interventions
Empagliflozin will be initiated at a dose of 0.3 mg/kg/dose (up to a maximum of 10 mg per dose), administered orally once daily before or after breakfast. Subsequent dosing may be adjusted according t
C570240
Sodium-Glucose Transporter 2 Inhibitors, empagliflozin
Sponsors
Akinari Fukuda
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients who have been diagnosed with GSDIb based on clinical findings or genetic testing prior to the screening period. 2. Patients with an ANC of less than 1,500 cells/mcL measured at the time of screening. 3. Patients with a confirmed ANC measurement within 12 months prior to the start of screening. 4. Patients aged 2 years or older and younger than 30 years at the time of consent. 5. Patients or their legally authorized representatives who have provided written informed consent.
Exclusion criteria
Exclusion criteria: 1. Patients with a history of hypersensitivity to empagliflozin. 2. Patients with severe ketosis, diabetic coma, or precoma. 3. Patients with a severe infection. 4. Patients with a serious traumatic injury. 5. Patients with end-stage renal disease (eGFR below 20 mL/min/1.73 m2) or undergoing dialysis. 6. Female patients who are pregnant, may be pregnant, or are breastfeeding. 7. Patients for whom discontinuation of G-CSF therapy prior to enrollment is deemed infeasible. 8. Patients with a history of SGLT2 inhibitor use within one week prior to the start of screening. 9. Patients deemed ineligible for the study by the principal investigator or a sub-investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in absolute neutrophil count (ANC) from baseline to the final day of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change from baseline to the final day of treatment for the following parameters 1) 1,5-Anhydroglucitol 2) Neutrophil function 2. Change from the final day of treatment to the last observation day for the following parameters 1) Absolute neutrophil count 2) 1,5-AG 3) Neutrophil function 3. Longitudinal changes from baseline to the last observation day for the following parameters and severity of oral mucositis: 1) ANC 2) 1,5-AG 3) Neutrophil function 4. Presence or absence of neutropenia on the final day of treatment 5. Incidence of all adverse events and all adverse drug reactions 6. Incidence of Grade 3 or more adverse events and adverse drug reactions 7. Proportion of time with blood glucose levels below 54 mg/dL from 3 days before to 4 days after treatment initiation | — |
Contacts
Public ContactYoshii Keisuke
National Center for Child Health and Development
Outcome results
None listed