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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
Children's Memorial Health Institute, Poland
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glucose 6 Phosphatase Deficiency

Eligibility Criteria

4 Weeks - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

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
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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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