Ignite Creation Date:
2024-05-06 @ 1:48 PM
Last Modification Date:
2024-10-26 @ 1:20 PM
Study NCT ID:
NCT04138251
Status:
UNKNOWN
Last Update Posted:
2019-10-28
First Post:
2019-06-20
Brief Title:
Safety Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency
Sponsor:
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Organization:
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Study Overview
Official Title:
Evaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia in Patients With Glycogenosis Type 1b and G6PC3 Deficiency
Status:
UNKNOWN
Status Verified Date:
2019-10
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
GLYCO-1B
Brief Summary:
Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin
Detailed Description:
Ubiquitous glucose-6-phosphatase deficiency G6PC3 and glucose-6-phosphate transporter deficiency G6PTSLC37A4 both cause neutropenia Studies on a G6PC3 deficient mouse model by Dr Veiga-da-Cunha and Prof Van Schaftingen and colleagues have shown that these two proteins collaborate to hydrolyze a metabolite that exerts toxic effects on neutrophils This metabolite is 15-anhydroglucitol-6-phosphate It is formed by phosphorylation of a glucose analogue 15-anhydroglucitol which is present in the blood of all humans mice and other mammals
This discovery of the function of G6PC3 and G6PT opens up therapeutic prospects in that lowering the concentration of 15-anhydroglucitol in the blood should reduce the concentration of 15-anhydroglucitol-6-phosphate in the cells and thus reduce its toxic effects Veiga-da-Cunha Van Schaftingen and colleagues have already shown that this is the case for a model of mice deficient in G6PC3 treated with empagliflozin
Following these discoveries the aim of the investigators experiment is to test the effect of the efficacy of empagliflozin on urinary excretion and elimination of blood 15-anhydroglucitol in patients with glucose-6-phosphate transporter deficiency type Ib glycogenosis and patients with G6PC3 deficiency This should allow patients to significantly lower the level of 15-anhydroglucitol-6-phosphate found in their neutrophils and thus cure their neutropenia
Empagliflozin marketed in Belgium under the name of Jardiance belongs to the class of drugs called oral hypoglycemic agents It works on the kidney by inhibiting the glucose transporter in the proximal tubules SGLT2 which leads to glucosuria that results in the elimination of 15-anhydroglucitol in the urine At present Empagliflozin alone or in combination with other drugs is commonly used in people with type 2 diabetes to control their blood sugar levels
This discovery of the function of G6PC3 and G6PT opens up therapeutic prospects in that lowering the concentration of 15-anhydroglucitol in the blood should reduce the concentration of 15-anhydroglucitol-6-phosphate in the cells and thus reduce its toxic effects Veiga-da-Cunha Van Schaftingen and colleagues have already shown that this is the case for a model of mice deficient in G6PC3 treated with empagliflozin
Following these discoveries the aim of the investigators experiment is to test the effect of the efficacy of empagliflozin on urinary excretion and elimination of blood 15-anhydroglucitol in patients with glucose-6-phosphate transporter deficiency type Ib glycogenosis and patients with G6PC3 deficiency This should allow patients to significantly lower the level of 15-anhydroglucitol-6-phosphate found in their neutrophils and thus cure their neutropenia
Empagliflozin marketed in Belgium under the name of Jardiance belongs to the class of drugs called oral hypoglycemic agents It works on the kidney by inhibiting the glucose transporter in the proximal tubules SGLT2 which leads to glucosuria that results in the elimination of 15-anhydroglucitol in the urine At present Empagliflozin alone or in combination with other drugs is commonly used in people with type 2 diabetes to control their blood sugar levels
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None