Back to resultsGalactose Supplementation for the Treatment of MOGHE (GATE)
Primary Purpose
Refractory Epilepsy, SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation
Status
Unknown status
Phase
Not Applicable
Locations
Spain
Study Type
Interventional
Intervention
D-Galactose
Sponsored by
About this trial
This is an interventional treatment trial for Refractory Epilepsy
Eligibility Criteria
Inclusion Criteria:
- MOGHE diagnosis at histopathological examination of the epilepsy surgery tissue.
- Epilepsy refractoriness or ongoing epileptiform activity at EEG.
Exclusion Criteria:
- Allergy to galactose or supplement components.
Sites / Locations
- Hospital Ruber InternacionalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Single arm group
Arm Description
Galactose supplementation
Outcomes
Primary Outcome Measures
Seizure frequency
Seizure diary
Epileptiform activity at EEG
Epileptiform activity quantification (per epoch)
Secondary Outcome Measures
Behavioral assessment
Teacher and Parent Rating Scale (SNAP-IV) and Conners Continuous Performance Test (CPT-II)
Cognitive assessment
Evaluated with Wechsler Intelligence Scale for Children (WISC-IV) and Behavior Rating Inventory of Executive Function-2 (BRIEF-2)
Full Information
NCT ID
NCT04833322
First Posted
March 31, 2021
Last Updated
April 3, 2021
Sponsor
Hospital Ruber Internacional
1. Study Identification
Unique Protocol Identification Number
NCT04833322
Brief Title
Galactose Supplementation for the Treatment of MOGHE
Acronym
GATE
Official Title
Galactose Supplementation for the Treatment of Patients With Mild Malformation of Cortical Development With Oligodendroglial Hyperplasia in Epilepsy (MOGHE): a Pilot Trial
Study Type
Interventional
2. Study Status
Record Verification Date
April 2021
Overall Recruitment Status
Unknown status
Study Start Date
January 15, 2021 (Actual)
Primary Completion Date
September 1, 2021 (Anticipated)
Study Completion Date
October 1, 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hospital Ruber Internacional
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Mild malformation of cortical development with oligodendroglial hyperplasia in epilepsy (MOGHE) is a new entity frequently associated with refractory epilepsy and neurodevelopmental disorders. Recently, it has been associated to SLC35A2 (Solute Carrier Family 35 Member A2) brain mosaic pathogenic variants. In addition, patients with germline SLC35A2 pathogenic variants improve with galactose supplementation. Therefore, the investigators aim to elucidate whether d-galactose as an add-on treatment might improve epilepsy and developmental outcomes in patients with MOGHE.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Refractory Epilepsy, SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Model Description
Before-and-after pilot clinical trial with a single group
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Single arm group
Arm Type
Experimental
Arm Description
Galactose supplementation
Intervention Type
Dietary Supplement
Intervention Name(s)
D-Galactose
Intervention Description
Galactose supplementation, once per day, up to 1.5g/kg per day
Primary Outcome Measure Information:
Title
Seizure frequency
Description
Seizure diary
Time Frame
6 months
Title
Epileptiform activity at EEG
Description
Epileptiform activity quantification (per epoch)
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Behavioral assessment
Description
Teacher and Parent Rating Scale (SNAP-IV) and Conners Continuous Performance Test (CPT-II)
Time Frame
6 months
Title
Cognitive assessment
Description
Evaluated with Wechsler Intelligence Scale for Children (WISC-IV) and Behavior Rating Inventory of Executive Function-2 (BRIEF-2)
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
MOGHE diagnosis at histopathological examination of the epilepsy surgery tissue.
Epilepsy refractoriness or ongoing epileptiform activity at EEG.
Exclusion Criteria:
Allergy to galactose or supplement components.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Angel Aledo-Serrano, MD PhD
Phone
0034-913875250
Email
aaledo@neurologiaclinica.es
First Name & Middle Initial & Last Name or Official Title & Degree
Ana Rodriguez
Phone
0034-913875250
Email
ensayosepi@neurologiaclinica.es
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Angel Aledo-Serrano, MD, PhD
Organizational Affiliation
Hospital Ruber Internacional
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital Ruber Internacional
City
Madrid
State/Province
Madrid, Comunidad De
ZIP/Postal Code
28034
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angel A Aledo-Serrano, MD PhD
Phone
0034-913875250
Email
aaledo@neurologiaclinica.es
First Name & Middle Initial & Last Name & Degree
Angel Aledo-Serrano, MD PhD
First Name & Middle Initial & Last Name & Degree
Antonio Gil-Nagel, MD PhD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Galactose Supplementation for the Treatment of MOGHE
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