EMPAtia: Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

Sponsor
Children's Memorial Health Institute, Poland (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT04930627
Collaborator
Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw (Other)
20
1
1
44
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Study Details

Study Description

Brief Summary

Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin

Condition or Disease Intervention/Treatment Phase
Phase 2

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 Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
Anticipated Study Start Date :
Jul 1, 2021
Anticipated Primary Completion Date :
Jun 1, 2024
Anticipated Study Completion Date :
Mar 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: oral administration of Empagliflozin

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 Names:
  • Jardiance
  • Outcome Measures

    Primary Outcome Measures

    1. Empaglifozin safety and tolerability measured by occurrence of adverse reactions [2 years]

      Empaglifozin saftey and tolerability measured by occurrence of adverse reactions

    Secondary Outcome Measures

    1. Efficacy of neutropenia treatment measured as percentage of the patients [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

    2. Dosis change/withdrawal of filgrastrim [2 years]

      Dosis change/withdrawal of filgrastrim

    3. Degree of metabolic compensation [2 years]

      measured as change of triglycerides (mg/dL), lactate (mg/dL), and uric acid (mg/dL) compared to the period before study

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Weeks and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 The Children's Memorial Health Institute Warsaw Poland 04-730

    Sponsors and Collaborators

    • Children's Memorial Health Institute, Poland
    • Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Dariusz Rokicki, Pricipal Investigator, Children's Memorial Health Institute, Poland
    ClinicalTrials.gov Identifier:
    NCT04930627
    Other Study ID Numbers:
    • EMPAtia
    • 2021-000580-78
    First Posted:
    Jun 18, 2021
    Last Update Posted:
    Jun 23, 2021
    Last Verified:
    Jun 1, 2021
    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
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Jun 23, 2021