Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia (EMPAtia)
Primary Purpose
Glucose 6 Phosphatase Deficiency
Status
Not yet recruiting
Phase
Phase 2
Locations
Poland
Study Type
Interventional
Intervention
Empagliflozin
Sponsored by
About this trial
This is an interventional treatment trial for Glucose 6 Phosphatase Deficiency
Eligibility Criteria
Inclusion Criteria:
- Minimum age 4 weeks old female Or Male
- GSD1b confirmed by genetic analysis with neutropenia and/or reduced respiratory burst
- Informed consent signed by the parents/assigns, and the recipient (>13 years old)
Exclusion Criteria:
- Risk of non-compliance
- Chronic renal diseases (eGFR < 60 ml/min/1,73 m2)
- Active urinary tract infection (temporal criterion, up to recovery)
- Participation in another clinical trial (minimum 6 months from the end of participation until the date of signing the Informed Consent Form)
- Participation in therapeutic experiment, in addition to the experimental treatment with empagliflozin (minimum 12 months from the end of participation until the date of signing the Informed Consent Form)
- Pregnancy, breastfeeding
- Allergy to Empagliflozin
- Lack of informed consent
Sites / Locations
- The Children's Memorial Health Institute
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
oral administration of Empagliflozin
Arm Description
Outcomes
Primary Outcome Measures
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
Secondary Outcome Measures
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
Dosis change/withdrawal of filgrastrim
Dosis change/withdrawal of filgrastrim
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
Full Information
NCT ID
NCT04930627
First Posted
June 11, 2021
Last Updated
June 18, 2021
Sponsor
Children's Memorial Health Institute, Poland
Collaborators
Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
1. Study Identification
Unique Protocol Identification Number
NCT04930627
Brief Title
Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia
Acronym
EMPAtia
Official Title
Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
Study Type
Interventional
2. Study Status
Record Verification Date
June 2021
Overall Recruitment Status
Not yet recruiting
Study Start Date
July 2021 (Anticipated)
Primary Completion Date
June 2024 (Anticipated)
Study Completion Date
March 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Memorial Health Institute, Poland
Collaborators
Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
5. Study Description
Brief Summary
Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin
Detailed Description
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®).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glucose 6 Phosphatase Deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
oral administration of Empagliflozin
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Empagliflozin
Other Intervention Name(s)
Jardiance
Intervention Description
dosis depending on body weight: <20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; >40 kg 2 x 10 mg
Primary Outcome Measure Information:
Title
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
Description
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Efficacy of neutropenia treatment measured as percentage of the patients
Description
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
Title
Dosis change/withdrawal of filgrastrim
Description
Dosis change/withdrawal of filgrastrim
Time Frame
2 years
Title
Degree of metabolic compensation
Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Weeks
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Minimum age 4 weeks old female Or Male
GSD1b confirmed by genetic analysis with neutropenia and/or reduced respiratory burst
Informed consent signed by the parents/assigns, and the recipient (>13 years old)
Exclusion Criteria:
Risk of non-compliance
Chronic renal diseases (eGFR < 60 ml/min/1,73 m2)
Active urinary tract infection (temporal criterion, up to recovery)
Participation in another clinical trial (minimum 6 months from the end of participation until the date of signing the Informed Consent Form)
Participation in therapeutic experiment, in addition to the experimental treatment with empagliflozin (minimum 12 months from the end of participation until the date of signing the Informed Consent Form)
Pregnancy, breastfeeding
Allergy to Empagliflozin
Lack of informed consent
Facility Information:
Facility Name
The Children's Memorial Health Institute
City
Warsaw
ZIP/Postal Code
04-730
Country
Poland
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Magdalena Kaczor, MD
Phone
+48227494
Email
mwojtylo@o2.pl
First Name & Middle Initial & Last Name & Degree
Dariusz Rokicki, MD PhD
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia
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