Glucose 6 Phosphatase DeficiencyPhase 2EMPAtiaNCT04930627
Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia
A Phase 2 study testing Empagliflozin in people with Glucose 6 Phosphatase Deficiency, run by Children's Memorial Health Institute, Poland at 1 site in Poland.
Trial summary
Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin Read more about this study
- Who is it for?
- Ages 4 weeks and older
- Conditions the study is for
- Glucose 6 Phosphatase Deficiency
- What is being tested?
- Empagliflozin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 sitePoland
- Study dates
- Jul 2021to Mar 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 weeks and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Poland1 site
The Children's Memorial Health Institute
Warsaw, 04-730
Magdalena Kaczor, MD, +48227494, mwojtylo@o2.pl
Dariusz Rokicki, MD PhD
About this study
Official title: Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin
Read the 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®).
- Conditions
- Glucose 6 Phosphatase Deficiency
- Being tested
- Empagliflozin
- Medical subject headings
- Glycogen Storage Disease Type I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmpagliflozinDrug
Also called Jardiance
dosis depending on body weight: <20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; >40 kg 2 x 10 mg
| Study group | Role | What they receive |
|---|---|---|
| oral administration of Empagliflozin | Experimental | Not described Receives: Drug: Empagliflozin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
Measured: 2 years
Secondary outcomes (3)
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
Measured: 2 years
Dosis change/withdrawal of filgrastrim
Dosis change/withdrawal of filgrastrim
Measured: 2 years
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
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2021
- Study startJul 2021 (estimated)
- Primary completionJun 2024 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glucose 6 Phosphatase DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Memorial Health Institute, Poland
- Sponsor type
- University, hospital or other
- Collaborators
- Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
- Responsible party
- Principal investigator, Dariusz Rokicki, Children's Memorial Health Institute, Poland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04930627
- Sponsor's protocol ID
- EMPAtia
- Other IDs
- 2021-000580-78
- Acronym
- EMPAtia
- Status last verified
- Jun 2021
- First submitted
- 11 Jun 2021
- Last updated
- 23 Jun 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
- ClinicalTrials.gov ID
- NCT04930627
- Acronym / sponsor ID
- EMPAtia / EMPAtia
- Conditions
- Glucose 6 Phosphatase Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Children's Memorial Health Institute, PolandUniversity, hospital or other
- Collaborators
- Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw
- Study start
- Jul 2021 (estimated)
- Primary completion
- Jun 2024 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 18 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| oral administration of Empagliflozin | Experimental | Drug: Empagliflozin |
Who may qualify
- Ages
- 4 weeks and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmpagliflozinAlso: Jardiancedosis depending on body weight: <20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; >40 kg 2 x 10 mg | Drug | oral administration of Empagliflozin |
Primary outcome
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
Time frame: 2 years
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
3 secondary outcomes
Efficacy of neutropenia treatment measured as percentage of the patients
Time frame: 2 years
Dosis change/withdrawal of filgrastrim
Time frame: 2 years
Degree of metabolic compensation
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Empagliflozin in people with Glucose 6 Phosphatase Deficiency, run by Children's Memorial Health Institute, Poland at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Empaglifozin safety and tolerability measured by occurrence of adverse reactions (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2021.