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Glycogen Storage Disease Type IPhase 2GLYCO-1BNCT04138251

Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency

A Phase 2 study testing Empagliflozin in people with Glycogen Storage Disease Type I and Glucose 6 Phosphatase Deficiency, run by Cliniques universitaires Saint-Luc- Université Catholique de Louvain at 1 site in Belgium.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin Read more about this study

Who is it for?
Ages 1 year to 18 years
What is being tested?
Empagliflozin
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
5 participants
Where
1 siteBelgium
Study dates
20 Jun 2019to 30 Jun 2020
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 1 year to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Glycogenosis type 1b confirmed by biochemical analyzes and / or genetic analysis. These patients with Glycogenosis must have had a liver transplant
  • Alternatively, G6PC3 deficiency confirmed by genetic analysis
  • Age 1 to 18 years old female or male
  • Informed consent signed by the recipient and / or parents / assigns.
  • Information and agreement of the referring medical team.
  • A Negative Blood Pregnancy Test at the time of screening and a negative urinary pregnancy test at Day 1 of the protocol are required for female with child bearing potential.
  • Sexually active patients should use an effective method of contraception throughout the duration of the study and up to 7 days after the last dose of Empaglifozine. (The combination of a hormonal method and a barrier method; Two barrier methods, the male condom being one of these two methods;Use intrauterine device or tubal ligation;-A total sex abstinence.)

Who cannot join

  • Presence of advanced fibrosis (Metavir F4) or cirrhosis.
  • Impossibility of long-term and / or non-compliance monitoring.
  • Other medical problems which, in the opinion of the physicians in charge of the patient, would constitute a contraindication to the procedure.
  • Sexually active patients who do not consent to use effective contraception during the study.

Trial locations and contacts

1 site in 1 country.

Xavier Stephenne, MD, PhD contact

32 2 7641377

xavier.stephenne@uclouvain.be

Julia Versavau contact

32 2 7641933

Belgium1 site, 1 recruiting

  • Cliniques universitaires Saint-Luc

    Brussels, 1200

    Xavier Stephenne, MD, PhD, 32 2 7641377, xavier.stephenne@uclouvain.be

    Xavier Stephenne, MD, PhD

    Recruiting

Other trials in Belgium

About this study

Official title: Evaluation of the Safety and Efficacy of Empagliflozin Administration as a Treatment for Neutropenia in Patients With Glycogenosis Type 1b and G6PC3 Deficiency

Treatment of neutropenia of G6PC3 and Glycogenosis type 1b patients with empagliflozin

Read the detailed description

Ubiquitous glucose-6-phosphatase deficiency (G6PC3) and glucose-6-phosphate transporter deficiency (G6PT/SLC37A4) both cause neutropenia. Studies on a G6PC3 deficient mouse model by Dr Veiga-da-Cunha and Prof. Van Schaftingen and colleagues have shown that these two proteins collaborate to hydrolyze a metabolite that exerts toxic effects on neutrophils. This metabolite is 1,5-anhydroglucitol-6-phosphate. It is formed by phosphorylation of a glucose analogue, 1,5-anhydroglucitol, which is present in the blood of all humans, mice and other mammals.

This discovery of the function of G6PC3 and G6PT opens up therapeutic prospects, in that lowering the concentration of 1,5-anhydroglucitol in the blood should reduce the concentration of 1,5-anhydroglucitol-6-phosphate in the cells and thus reduce its toxic effects. Veiga-da-Cunha, Van Schaftingen and colleagues have already shown that this is the case for a model of mice deficient in G6PC3 treated with empagliflozin .

Following these discoveries, the aim of the investigator's experiment is to test the effect of the efficacy of empagliflozin on urinary excretion and elimination of blood 1,5-anhydroglucitol in patients with glucose-6-phosphate transporter deficiency (type Ib glycogenosis) and patients with G6PC3 deficiency. This should allow patients to significantly lower the level of 1,5-anhydroglucitol-6-phosphate found in their neutrophils and thus cure their neutropenia.

Empagliflozin (marketed in Belgium under the name of Jardiance®) belongs to the class of drugs called oral hypoglycemic agents. It works on the kidney by inhibiting the glucose transporter in the proximal tubules, SGLT2, which leads to glucosuria that results in the elimination of 1,5-anhydroglucitol in the urine. At present, Empagliflozin alone or in combination with other drugs is commonly used in people with type 2 diabetes to control their blood sugar levels.

Being tested
Empagliflozin
Keywords
Empaglifozin
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

    oral administration of Empagliflozin

Study groups in NCT04138251
Study groupRoleWhat they receive
TreatmentExperimentalOral empaglifozin 5 mg 1x/day, increase up to 10 mg 1x/day if no 25% decrease of blood1,5-anhydroglucitol level
Receives: Drug: Empagliflozin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Empaglifozin safety (blood test-glycemia): measured by absence of hypoglycaemia due to gliflozin treatment

    Empaglifozin safety is measured by absence of hypoglycaemia due to gliflozin treatment (continuous monitoring during the first 2 days of treatment and glycemia punctual monitoring every 7 days for 2 months) (mg/dl)

    Measured: from start of treatment to 2 months post treatment

  2. Empaglifozin Efficacy (blood test-hemogram)

    Efficacy of drug is measured by an Increased neutrophil count as compared to pre-treatment (10exp3/µl)

    Measured: from start of treatment to 2 months post treatment

Secondary outcomes (5)

  1. Empaglifozin Clinical efficacy (questionnaire)

    Empaglifozin Clinical efficacy is measured as a Decrease in the number of infections

    -Decrease in the number of episodes of oral aphtosis (stomatitis) We will use numerical scale: higher scores mean worse outcome

    Measured: from start of treatment to 2 months post treatment

  2. Empaglifozin Biological efficacy on blood 1,5-anhydroglucitol level (blood test-LCMS)

    Empaglifozin Biological efficacy is measured as a Decrease of blood 1,5-anhydroglucitol (µM)

    Measured: from start of treatment to 2 months post treatment

  3. Empaglifozin Biological efficacy on 1,5-anhydroglucitol-6-phosphate levels in neutrophils (blood test-LCMS)

    Empaglifozin Biological efficacy is measured as decrease in the level of 1,5-anhydroglucitol-6-phosphate in neutrophils (µM)

    Measured: from start of treatment to 2 months post treatment

  4. Empaglifozin Clinical efficacy on urinary 1,5-anhydroglucitol excretion increase (urine test-LCMS)

    Empaglifozin Biological efficacy is measured as increased excretion of urinary 1,5-anhydroglucitol (µM)

    Measured: from start of treatment to 2 months post treatment

  5. Empaglifozin Clinical efficacy on neutrophil function (blood test)

    Empaglifozin Biological efficacy is measured as improved neutrophilic function (glycosylation analysis, Western Blot)

    Measured: from start of treatment to 2 months post treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Oct 2019
  2. Study start20 Jun 2019
  3. Primary completion30 Jun 2020 (estimated)
  4. Study completion30 Jun 2020 (estimated)

Resources

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
5 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04138251
Sponsor's protocol ID
2018/26DEC/492
Acronym
GLYCO-1B
Status last verified
Oct 2019
First submitted
20 Jun 2019
Last updated
28 Oct 2019
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Empagliflozin in people with Glycogen Storage Disease Type I and Glucose 6 Phosphatase Deficiency, run by Cliniques universitaires Saint-Luc- Université Catholique de Louvain at 1 site in Belgium.

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 7 inclusion and 4 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 28 Oct 2019. 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 Belgium. 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?

Contact Xavier Stephenne, MD, PhD (32 2 7641377, xavier.stephenne@uclouvain.be), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Empaglifozin safety (blood test-glycemia): measured by absence of hypoglycaemia due to gliflozin treatment (time frame: from start of treatment to 2 months post treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04138251.

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 28 Oct 2019.

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