GLUT1 Deficiency SyndromeEarly Phase 1NCT04112862
Sodium Lactate Infusion in GLUT1DS Patients
A Early Phase 1 study testing sodiumlactate in people with GLUT1 Deficiency Syndrome and GLUT1DS1, run by Radboud University Medical Center at 1 site in Netherlands.
Trial summary
This study investigates the effect of lactate infusion on epileptic discharges on EEG and seizure frequency in glucose transporter 1 deficiency syndrome (GLUT1DS) patients. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- GLUT1 Deficiency Syndrome, GLUT1DS1, GLUT1DS2
- What is being tested?
- sodiumlactate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteNetherlands
- Study dates
- 20 May 2022to 18 Nov 2022
- Phase
- Early Phase 1
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with GLUT1DS and known in our center.
- Baseline characteristics include a high frequency of clinical seizures and epileptic discharges on EEG.
- History of trying ketogenic diet with good compliance without beneficial effects.
- Age > 6 years.
- Informed consent.
Who cannot join
- Additional medical condition or illness that impairs the patient's ability to participate in the study (for example actual treatment of a malignancy, active infection, poorly controlled diabetes mellitus, hypertension, organ failure, clinically significant haematological or biochemical abnormalities).
- Elevated serum sodium (> 145 mmol/L).
- Participation in another interventional study at start of the study or during the study.
- Presence of known panic disorders or a history of panic attacks.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effects of Sodium Lactate Infusion in Patients With Glucose Transporter 1 Deficiency Syndrome (GLUT1DS)
This study investigates the effect of lactate infusion on epileptic discharges on EEG and seizure frequency in glucose transporter 1 deficiency syndrome (GLUT1DS) patients.
Read the detailed description
In glucose transporter 1 deficiency syndrome (GLUT1DS) cerebral glucose uptake from the systemic blood circulation is limited, because of deficient transport of glucose across the blood-brain barrier by the transporter protein (GLUT1). Classically patients present with developmental problems, movement disorders and severe epilepsy. There is no curative treatment for GLUT1DS, and anti-epileptic drugs usually have little to no effect. The ketogenic diet, providing ketones as an alternative energy substrate for the brain is an effective treatment option for the epilepsy and movement disorders in many GLUT1DS patients. Unfortunately, not in all GLUT1DS patients the ketogenic diet has a positive effect and other treatment options for these patients are very limited.
Traditionally, lactate is seen as a waste product of glycolysis during anaerobic conditions and a marker of ischemia. Interestingly, research has shown the beneficial side of lactate as an energy source for different organs. It seems that the brain can indeed use lactate as an alternative energy metabolite, besides glucose and ketones. The aim of this study is to investigate whether lactate can be an alternative energy metabolite in the GLUT1DS brain, and reduce epileptic discharges on EEG when intravenously administered in patients with GLUT1DS.
- Conditions
- GLUT1 Deficiency Syndrome, GLUT1DS1, GLUT1DS2, Epilepsy, Lactic Acid Blood Increased, Ketogenic Dieting
- Being tested
- sodiumlactate
- Keywords
- GLUT1 deficiency syndrome, sodium lactate infusion, epilepsy
- Medical subject headings
- Glut1 Deficiency Syndrome, Dystonia 18, Epilepsy, Hyperlactatemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sodiumlactateOther
intravenous sodiumlactate 600mmol/L
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | three GLUT1DS patients with drug resistant epilepsy who tried the ketogenic diet without any benefits. Receives: Other: sodiumlactate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of changes in epileptic discharges on EEG during the infusion of lactate.
All subjects have frequent epileptic discharges on EEG. We want to objectify the change in epileptic discharges on EEG during and after the infusion of sodium lactate.
Measured: 1 day
Secondary outcomes (8)
Change in seizure frequency clinical visible by caregivers during and after infusion of sodium lactate.
All subjects have frequent seizures, we want to see if these clinically visible seizures reduce during or after infusion with sodium lactate.
Measured: 1 day
Concentration of lactate in blood during and after sodium lactate infusion
Measured: 1 day
Concentration of glucose in blood during and after sodium lactate infusion
Measured: 1 day
Concentration of sodium in blood during and after sodium lactate infusion
Measured: 1 day
Concentration of potassium in blood during and after sodium lactate infusion
Measured: 1 day
Concentration of bicarbonate in blood during and after sodium lactate infusion
Measured: 1 day
Concentration of chloride in blood during and after sodium lactate infusion
Measured: 1 day
pH in blood during and after sodium lactate infusion
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2019
- Study start20 May 2022
- Primary completion18 Nov 2022
- Study completion18 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GLUT1 Deficiency SyndromeStudies of the same condition, open and under way.
- More trials run by Radboud University Medical CenterThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single center, interventional, explorative, open label, proof of principle study
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Radboud University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04112862
- Sponsor's protocol ID
- 109147
- Status last verified
- Feb 2022
- First submitted
- 18 Sep 2019
- Last updated
- 23 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: We want to include three patients, who all are there own control. If one of their EEGs shows interesting changes during or after lactate infusion, we plan to describe that in our article.
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Sodium Lactate Infusion in Patients With Glucose Transporter 1 Deficiency Syndrome (GLUT1DS)
- ClinicalTrials.gov ID
- NCT04112862
- Acronym / sponsor ID
- 109147
- Conditions
- GLUT1 Deficiency Syndrome, GLUT1DS1, GLUT1DS2, Epilepsy, Lactic Acid Blood Increased, Ketogenic Dieting
- Keywords
- GLUT1 deficiency syndrome, sodium lactate infusion, epilepsy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Radboud University Medical CenterUniversity, hospital or other
- Study start
- 20 May 2022 (actual)
- Primary completion
- 18 Nov 2022 (actual)
- Study completion
- 18 Nov 2022 (actual)
- First posted
- 2 Oct 2019 (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.
This study investigates the effect of lactate infusion on epileptic discharges on EEG and seizure frequency in glucose transporter 1 deficiency syndrome (GLUT1DS) patients.
Detailed description
In glucose transporter 1 deficiency syndrome (GLUT1DS) cerebral glucose uptake from the systemic blood circulation is limited, because of deficient transport of glucose across the blood-brain barrier by the transporter protein (GLUT1). Classically patients present with developmental problems, movement disorders and severe epilepsy. There is no curative treatment for GLUT1DS, and anti-epileptic drugs usually have little to no effect. The ketogenic diet, providing ketones as an alternative energy substrate for the brain is an effective treatment option for the epilepsy and movement disorders in many GLUT1DS patients. Unfortunately, not in all GLUT1DS patients the ketogenic diet has a positive effect and other treatment options for these patients are very limited.
Traditionally, lactate is seen as a waste product of glycolysis during anaerobic conditions and a marker of ischemia. Interestingly, research has shown the beneficial side of lactate as an energy source for different organs. It seems that the brain can indeed use lactate as an alternative energy metabolite, besides glucose and ketones. The aim of this study is to investigate whether lactate can be an alternative energy metabolite in the GLUT1DS brain, and reduce epileptic discharges on EEG when intravenously administered in patients with GLUT1DS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single center, interventional, explorative, open label, proof of principle study
| Arm | Type | Receives |
|---|---|---|
| Intervention | Experimental | Other: sodiumlactatethree GLUT1DS patients with drug resistant epilepsy who tried the ketogenic diet without any benefits. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Diagnosed with GLUT1DS and known in our center.
- Baseline characteristics include a high frequency of clinical seizures and epileptic discharges on EEG.
- History of trying ketogenic diet with good compliance without beneficial effects.
- Age > 6 years.
- Informed consent.
Key exclusion criteria (4 listed)
- Additional medical condition or illness that impairs the patient's ability to participate in the study (for example actual treatment of a malignancy, active infection, poorly controlled diabetes mellitus, hypertension, organ failure, clinically significant haematological or biochemical abnormalities).
- Elevated serum sodium (> 145 mmol/L).
- Participation in another interventional study at start of the study or during the study.
- Presence of known panic disorders or a history of panic attacks.
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 |
|---|---|---|
| sodiumlactateintravenous sodiumlactate 600mmol/L | Other | Intervention |
Primary outcome
Number of changes in epileptic discharges on EEG during the infusion of lactate.
Time frame: 1 day
All subjects have frequent epileptic discharges on EEG. We want to objectify the change in epileptic discharges on EEG during and after the infusion of sodium lactate.
8 secondary outcomes
Change in seizure frequency clinical visible by caregivers during and after infusion of sodium lactate.
Time frame: 1 day
Concentration of lactate in blood during and after sodium lactate infusion
Time frame: 1 day
Concentration of glucose in blood during and after sodium lactate infusion
Time frame: 1 day
Concentration of sodium in blood during and after sodium lactate infusion
Time frame: 1 day
Concentration of potassium in blood during and after sodium lactate infusion
Time frame: 1 day
Concentration of bicarbonate in blood during and after sodium lactate infusion
Time frame: 1 day
Concentration of chloride in blood during and after sodium lactate infusion
Time frame: 1 day
pH in blood during and after sodium lactate infusion
Time frame: 1 day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2022.
- Individual participant dataSharing planned: No. We want to include three patients, who all are there own control. If one of their EEGs shows interesting changes during or after lactate infusion, we plan to describe that in our article.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing sodiumlactate in people with GLUT1 Deficiency Syndrome and GLUT1DS1, run by Radboud University Medical Center at 1 site in Netherlands.
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 5 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: finished, as of the sponsor's last update on 23 Nov 2022. 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 Netherlands. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of changes in epileptic discharges on EEG during the infusion of lactate. (time frame: 1 day). 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 Nov 2022.