NCT03722212

Brief Summary

The study aims at validating the diagnostic performances of the METAglut1, a blood in vitro diagnostic test, for the simple and early diagnosis of the Glut1 deficiency syndrome (Glut1DS, or De Vivo disease). The blood test will be carried out prospectively on patients presenting with a clinical suspicion of Glut1DS, blindly from the reference strategy, which consists in a lumbar puncture for glycorrhachia measurement, completed by a molecular analysis. The study will be conducted in more than 40 centers in France on up to 3,000 patients for 2 years.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
636

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started Sep 2018

Typical duration for not_applicable

Geographic Reach
1 country

28 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 24, 2018

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

October 25, 2018

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 26, 2018

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2021

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 9, 2021

Completed
Last Updated

December 1, 2022

Status Verified

August 1, 2022

Enrollment Period

2.5 years

First QC Date

October 25, 2018

Last Update Submit

November 28, 2022

Conditions

Keywords

Glut1 Deficiency SyndromeGlut1DS

Outcome Measures

Primary Outcomes (1)

  • Concordance analysis between METAglut1 and glycorrhachia

    This analysis will be performed on patients with a diagnosis of certainty, either positive or negative, in the prospective cohort. For this analysis two subgroups are distinguished: * Patients with lumbar puncture, molecular analysis of the slc2a1 gene and METAglut1 testing. * Patients with at least lumbar puncture and METAglut1 testing.

    Up to 6 months

Secondary Outcomes (1)

  • Sensitivity, specificity, positive and negative predictive values of METAglut1

    Up to 6 months

Other Outcomes (5)

  • Sensitivity, specificity of METAglut1

    Up to 6 moths

  • Concordance analysis between METAglut1 and glycorrhachia

    Up to 6 months

  • Sensitivity analysis with optimized threshold of positivity of the METAglut1™ assay

    Up to 6 months

  • +2 more other outcomes

Study Arms (2)

Prospective patients

OTHER

The METAglut1 test is performed on all patients included in the study. In parallel, patients included prospectively (based on a clinical suspicion) benefit from the reference diagnostic strategy through the current practice, starting with a lumbar puncture for glycorrhachia dosage.

Diagnostic Test: METAglut1

Retrospective patients

OTHER

Patients with confirmed Glut1DS diagnosis Already diagnosed patients are included retrospectively as well as patients with pending diagnosis at inclusion (inconsistent biological or genetic data).

Diagnostic Test: METAglut1

Interventions

METAglut1DIAGNOSTIC_TEST

A blood draw is performed on each patient for the METAglut1 test, and sent to Laboratoire CERBA, Saint-Ouen l'Aumône, France, for sample analysis.

Prospective patientsRetrospective patients

Eligibility Criteria

Age3 Months+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Clinical suspicion of the GLUT1 Deficiency Syndrome
  • Patients with confirmed Glut1DS diagnosis

You may not qualify if:

  • Patients under 3 months of age
  • Sickle cell disease S/S
  • Abnormal imaging

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

Hôpital Larrey- CHU Angers

Angers, 49933, France

Location

Hôpital Saint Léon

Bayonne, 64109, France

Location

Hôpital Jean Verdier- APHP

Bondy, 93140, France

Location

Centre hospitalier Pellegrin_ CHU Bordeaux

Bordeaux, 33076, France

Location

Hôpital Femme Mere enfant- CHU de Lyon

Bron, 69677, France

Location

Hospices Civils de Lyon_CHU Lyon

Bron, France

Location

Hôpital d'Estaing- CHU Clermont-Ferrand

Clermont-Ferrand, 63003, France

Location

CHU Dijon Bourgogne

Dijon, 21079, France

Location

Hôpital Raymond Poincaré- APHP

Garches, France

Location

Hôpital Nord_CHU Grenoble

La Tronche, 38700, France

Location

Hôpital Jeanne de Flandre _CHRU Lille

Lille, France

Location

Hôpital de la mère et de l'enfant- CHU Limoges

Limoges, 87042, France

Location

Hôpital La Timone Enfant- APHM

Marseille, 13385, France

Location

CHR Metz-Thionville

Metz, 57085, France

Location

Hôpital Gui de Chauliac- CHU Montpellier

Montpellier, 34295, France

Location

Hôpital Mère-Enfant_ CHU de Nantes

Nantes, France

Location

Hôpital la Pitié-Salpêtrière-APHP

Paris, 75013, France

Location

Hôpital Necker- APHP

Paris, 75015, France

Location

Hôpital Robert Debré- APHP

Paris, 75019, France

Location

Hôpital Trousseau- APHP

Paris, 75571, France

Location

Hôpital Bicêtre- APHP

Paris, France

Location

Hôpital Sud de Rennes- CHU Rennes

Rennes, 35203, France

Location

Hôpital de Saint-Nazaire

Saint-Nazaire, 44606, France

Location

Hôpital Nord, CHU Saint-Etienne

Saint-Priest-en-Jarez, 42270, France

Location

Hôpital de Hautepierre- CHU Strasbourg

Strasbourg, France

Location

Hôpital de Tarbes - CH Bigorre

Tarbes, 65013, France

Location

Hôpital des Enfants- CHU Toulouse

Toulouse, 31059, France

Location

Hôpital de Clocheville_ CHU Tours

Tours, 37000, France

Location

Related Publications (1)

  • Gras D, Cousin C, Kappeler C, Fung CW, Auvin S, Essid N, Chung BH, Da Costa L, Hainque E, Luton MP, Petit V, Vuillaumier-Barrot S, Boespflug-Tanguy O, Roze E, Mochel F. A simple blood test expedites the diagnosis of glucose transporter type 1 deficiency syndrome. Ann Neurol. 2017 Jul;82(1):133-138. doi: 10.1002/ana.24970.

    PMID: 28556183BACKGROUND

Related Links

MeSH Terms

Conditions

Glut1 Deficiency SyndromeSeizuresMovement DisordersIntellectual DisabilityAtaxia

Condition Hierarchy (Ancestors)

Neurologic ManifestationsNervous System DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsCentral Nervous System DiseasesNeurobehavioral ManifestationsNeurodevelopmental DisordersMental DisordersDyskinesias

Study Officials

  • Fanny Mochel, MD, PhD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Masking Details
The METAglut1 test is performed blindly of the reference strategy. METAglut1's result masking is maintained for the investigator on the one hand, and the glycorrhachia value masking is maintained for the centralized testing laboratory in charge of METAglut1 on the other hand.
Purpose
DIAGNOSTIC
Intervention Model
PARALLEL
Model Details: The METAglut1 test is performed on each patient, in parallel and blindly of the reference diagnostic strategy which is performed through the current practice (lumbar puncture (LP) performed in the fasting state with glycemia measured right before LP, followed by genetic analyses (targeted slc2a1 analysis or gene panels or whole exome sequencing).
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 25, 2018

First Posted

October 26, 2018

Study Start

September 24, 2018

Primary Completion

March 31, 2021

Study Completion

July 9, 2021

Last Updated

December 1, 2022

Record last verified: 2022-08

Data Sharing

IPD Sharing
Will not share

The data that support the findings of this study are available from the corresponding authors on reasonable request. Permissions are required to gain access to the data resources, subject to successful registration and application process.

Locations