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Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia (EMPAtia)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04930627
Recruitment Status : Not yet recruiting
First Posted : June 18, 2021
Last Update Posted : June 23, 2021
Sponsor:
Collaborator:
Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
Information provided by (Responsible Party):
Dariusz Rokicki, Children's Memorial Health Institute, Poland

Tracking Information
First Submitted Date  ICMJE June 11, 2021
First Posted Date  ICMJE June 18, 2021
Last Update Posted Date June 23, 2021
Estimated Study Start Date  ICMJE July 2021
Estimated Primary Completion Date June 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 11, 2021)		 Empaglifozin safety and tolerability measured by occurrence of adverse reactions [ Time Frame: 2 years ]
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 18, 2021)
  • Efficacy of neutropenia treatment measured as percentage of the patients [ Time Frame: 2 years ]
    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 [ Time Frame: 2 years ]
    Dosis change/withdrawal of filgrastrim
  • Degree of metabolic compensation [ Time Frame: 2 years ]
    measured as change of triglycerides (mg/dL), lactate (mg/dL), and uric acid (mg/dL) compared to the period before study
Original Secondary Outcome Measures  ICMJE
 (submitted: June 11, 2021)
  • Efficacy of neutropenia treatment measured as percentage of the patients [ Time Frame: 2 years ]
    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 reduction/withdrawal of filgrastrim [ Time Frame: 2 years ]
    Dosis reduction/withdrawal of filgrastrim
  • Degree of metabolic compensation [ Time Frame: 2 years ]
    measured as decrease of triglycerides, lactate, and uric acid compared to the period before study
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia
Official Title  ICMJE Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
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®).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Glucose 6 Phosphatase Deficiency
Intervention  ICMJE 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
Other Name: Jardiance
Study Arms  ICMJE Experimental: oral administration of Empagliflozin
Intervention: Drug: Empagliflozin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: June 11, 2021)		 20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2025
Estimated Primary Completion Date June 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Weeks and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Listed Location Countries  ICMJE Poland
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04930627
Other Study ID Numbers  ICMJE EMPAtia
2021-000580-78 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Dariusz Rokicki, Children's Memorial Health Institute, Poland
Original Responsible Party Dariusz Rokicki, Children's Memorial Health Institute, Poland, MD PhD
Current Study Sponsor  ICMJE Children's Memorial Health Institute, Poland
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
Investigators  ICMJE Not Provided
PRS Account Children's Memorial Health Institute, Poland
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP