A Phase 2 interventional study of Empagliflozin in Glucose 6 Phosphatase Deficiency, sponsored by Children's Memorial Health Institute, Poland. Status unknown at 1 site in Poland. Open to participants aged 4 Weeks and older. Per ClinicalTrials.gov, last updated 2021-06-23.
Sponsored by Children's Memorial Health Institute, Poland · Phase 2, Interventional, and Treatment
Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin
Symptoms of glycogen storage disease type Ib (GSD Ib) include - among others - hypoglycemia, hepatomegaly and neutropenia with concomitant neutrophil dysfunction, which results in recurrent bacterial and fungal infections, and inflammatory bowel disease.
At present filgrastim is the only available drug to treat neutropenia in GSD Ib patients; it stimulates neutrophil production, but doesn't restore their function. Part of GSD Ib patients doesn't respond to filgrastim treatment. The latest research results showed, that neutropenia and neutrophil dysfunction in GSD Ib patients are results of extensive accumulation of 1,5-anhydroglucitol-phosphate. Empagliflozin, a SGLT2 inhibitor, inhibits renal glucose and 1,5-anhydroglucitol reabsorption and is an effective and safe method of treatment of neutropenia in this group of patients. Empagliflozin (Jardiance®) is a drug, which is registered in Poland to treat type II diabetes in adults. The aim of our study is to evaluate the efficacy and safety of neutropenia in patients with GSD Ib with empagliflozin (Jardiance®).
Exclusion Criteria:
Drug: Empagliflozin
dosis depending on body weight: \<20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; \>40 kg 2 x 10 mg
Also known as: Jardiance
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
Time frame: 2 years
Efficacy of neutropenia treatment measured as percentage of the patients
who achieved \>500 neutrophils/ml for at least 6 months with normalization of oxidative burst with decrease of bacterial and fungal infections compared to the period before study with decrease of hospitalization number with decrease of the number of defecation, gingival sores, and calprotectin average concentration in stool
Time frame: 2 years
Dosis change/withdrawal of filgrastrim
Dosis change/withdrawal of filgrastrim
Time frame: 2 years
Degree of metabolic compensation
measured as change of triglycerides (mg/dL), lactate (mg/dL), and uric acid (mg/dL) compared to the period before study
Time frame: 2 years
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Children's Memorial Health Institute, Poland