Clinical Trial Page

Summary
EudraCT Number:2019-003676-39
Sponsor's Protocol Code Number:109147
National Competent Authority:Netherlands - Competent Authority
Clinical Trial Type:EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:2020-06-09
Trial results
Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL
A. Protocol Information
A.1Member State ConcernedNetherlands - Competent Authority
A.2EudraCT number2019-003676-39
A.3Full title of the trial
Effects of sodium lactate infusion in patients with glucose transporter 1 deficiency syndrome (GLUT1DS)
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
Lactate infusion in GLUT1DS
A.4.1Sponsor's protocol code number109147
A.7Trial is part of a Paediatric Investigation Plan No
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleTest
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation Yes
D.2.1.1.1Trade name sodiumlactate
D.2.1.2Country which granted the Marketing AuthorisationNetherlands
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.4Pharmaceutical form Powder for solution for infusion
D.3.4.1Specific paediatric formulation No
D.3.7Routes of administration for this IMPIntravenous use
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin Yes
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) No
D.3.11.3.1Somatic cell therapy medicinal product No
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product No
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another type of medicinal product No
D.8 Information on Placebo
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
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.
E.1.1.1Medical condition in easily understood language
GLUT1DS is a genetic syndrome characterized by developmental problems, movement disorders and severe epilepsy.
E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
MedDRA Classification
E.1.3Condition being studied is a rare disease No
E.2 Objective of the trial
E.2.1Main objective of the trial
The purpose of this explorative study is to investigate whether treatment with lactate has a positive effect on the drug-resistant epilepsy and EEG of patients with GLUT1DS.
The primary objective is to assess changes in EEG during and shortly after infusion of lacate.
E.2.2Secondary objectives of the trial
To assess any changes in seizure frequency during and shortly after infusion of lactate.
To assess any change in laboratory parameters during infusion and shortly after: lactate, pH, sodium, bicarbonate, chloride, potassium, glucose
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
In order to be eligible to participate in this study, a subject must meet all of the following criteria:
- 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 but without beneficial effects.
- Age > 6 years.
- Informed consent.
E.4Principal exclusion criteria
A potential subject who meets any of the following criteria will be excluded from participation in this study:
- 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.
E.5 End points
E.5.1Primary end point(s)
Change in frequency or form of epileptic discharges on EEG during of shortly after infusion of sodiumlactate.
The change in EEG will be measured by a neurologist experienced in reading EEG’s
E.5.1.1Timepoint(s) of evaluation of this end point
EEG will be continuously registered during the experiment.
E.5.2Secondary end point(s)
Seizure frequency: Subjects will be recorded on video camera and seizures will be monitored and counted by researchers and parents/caregivers.
Laboratory measures: All measurements will be performed in blood samples, and all samples will be analysed in laboratories of the Radboudumc.
- lactate concentration
- pH
- glucose
- sodium
- potassium
- chloride
- bicarbonate
E.5.2.1Timepoint(s) of evaluation of this end point
Seizure frequency: Subjects will be monitored continuously during the experiment.
Laboratory measures: T-60, T10, T20, T40, T60, T100, T120, T140, T200 and T240 minutes. Blood samples will be stopped earlier if all values have returned to baseline before the end of the experiment.
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis No
E.6.3Therapy Yes
E.6.4Safety No
E.6.5Efficacy Yes
E.6.6Pharmacokinetic Yes
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others No
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) No
E.7.1.1First administration to humans No
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) Yes
E.7.3Therapeutic confirmatory (Phase III) No
E.7.4Therapeutic use (Phase IV) No
E.8 Design of the trial
E.8.1Controlled Yes
E.8.1.1Randomised No
E.8.1.2Open Yes
E.8.1.3Single blind No
E.8.1.4Double blind No
E.8.1.5Parallel group No
E.8.1.6Cross over No
E.8.1.7Other Yes
E.8.1.7.1Other trial design description
Single center, interventional, explorative, open label, proof of principle study
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other Yes
E.8.2.3.1Comparator description
Each subject baseline characteristics are control of the subject.
E.8.2.4Number of treatment arms in the trial1
E.8.3 The trial involves single site in the Member State concerned Yes
E.8.4 The trial involves multiple sites in the Member State concerned No
E.8.5The trial involves multiple Member States No
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA No
E.8.6.2Trial being conducted completely outside of the EEA No
E.8.7Trial has a data monitoring committee No
E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
LVLS
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years
E.8.9.1In the Member State concerned months
E.8.9.1In the Member State concerned days1
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 Yes
F.1.1Number of subjects for this age range: 4
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) Yes
F.1.1.5.1Number of subjects for this age range: 4
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) No
F.1.3Elderly (>=65 years) No
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients Yes
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception No
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally Yes
F.3.3.6.1Details of subjects incapable of giving consent
Children who are under aged and possibly have cognitive impairment.
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state4
F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
Treatment and care after the trial will be normal treatment and care for GLUT1DS patients. Sodium lactate infusion will not be given after the trial.
G. Investigator Networks to be involved in the Trial
N. Review by the Competent Authority or Ethics Committee in the country concerned
N.Competent Authority Decision Authorised
N.Date of Competent Authority Decision2020-06-09
N.Ethics Committee Opinion of the trial application
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion
P. End of Trial
P.End of Trial Status

Sponsors and Collaborators

Medical Conditions

Drug Interventions

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