Condition or disease | Intervention/treatment | Phase |
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Glucose 6 Phosphatase Deficiency | Drug: Empagliflozin | Phase 2 |
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 : | Interventional (Clinical Trial) |
Estimated 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 |
Estimated Study Start Date : | July 2021 |
Estimated Primary Completion Date : | June 2024 |
Estimated Study Completion Date : | March 2025 |
Arm | Intervention/treatment |
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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 Name: Jardiance
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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Poland | |
The Children's Memorial Health Institute | |
Warsaw, Poland, 04-730 | |
Contact: Magdalena Kaczor, MD +48227494 mwojtylo@o2.pl | |
Principal Investigator: Dariusz Rokicki, MD PhD |
Tracking Information | |||||||
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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 |
Empaglifozin safety and tolerability measured by occurrence of adverse reactions [ Time Frame: 2 years ] Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
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Original Primary Outcome Measures ICMJE | Same as current | ||||||
Change History | |||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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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®). |
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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 |
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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
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Study Arms ICMJE | Experimental: oral administration of Empagliflozin
Intervention: Drug: Empagliflozin
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Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Not yet recruiting | ||||||
Estimated Enrollment ICMJE |
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:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 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 ) |
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Has Data Monitoring Committee | Not Provided | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||||
Responsible Party | Dariusz Rokicki, Children's Memorial Health Institute, Poland | ||||||
Study Sponsor ICMJE | Children's Memorial Health Institute, Poland | ||||||
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 |