Galactose Supplementation for the Treatment of MOGHE | NCT04833322

Studies
Galactose Supplementation for the Treatment of MOGHE

NCT04833322

Sponsor

Hospital Ruber Internacional

Status

Unknown status

Last Update Posted

Apr 6, 2021Actual

Enrollment

8Estimated

Phase

Not Applicable

Conditions

Refractory Epilepsy
SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation

Interventions

D-Galactose

Countries

Spain

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2021

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 15, 2021Actual

Primary Completion Date

Sep 1, 2021Estimated

Completion Date

Oct 1, 2021Estimated

First Submitted Date

Mar 31, 2021

First Submission Date that Met QC Criteria

Apr 3, 2021

First Posted Date

Apr 6, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 3, 2021

Last Update Posted Date

Apr 6, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Angel Aledo-Serrano, Principal Investigator, Hospital Ruber InternacionalPrincipal Investigator

Lead Sponsor

Hospital Ruber InternacionalOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

8Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Single arm group	Experimental	Galactose supplementation	Dietary Supplement: D-Galactose

Interventions

Name	Type	Description	Arm Group Labels	Other Names
D-Galactose	Dietary Supplement	Galactose supplementation, once per day, up to 1.5g/kg per day	Single arm group

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Seizure frequency	Seizure diary	6 months
Epileptiform activity at EEG	Epileptiform activity quantification (per epoch)	6 months

Secondary Outcomes

Measure	Description	Time Frame
Behavioral assessment	Teacher and Parent Rating Scale (SNAP-IV) and Conners Continuous Performance Test (CPT-II)	6 months
Cognitive assessment	Evaluated with Wechsler Intelligence Scale for Children (WISC-IV) and Behavior Rating Inventory of Executive Function-2 (BRIEF-2)

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

20 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Angel Aledo-Serrano, MD PhD	Contact	0034-913875250		aaledo@neurologiaclinica.es
Ana Rodriguez	Contact	0034-913875250		ensayosepi@neurologiaclinica.es

Overall Officials

Name	Affiliation	Role
Angel Aledo-Serrano, MD, PhD	Hospital Ruber Internacional	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Ruber Internacional	Recruiting	Madrid	Madrid, Comunidad de	28034	Spain

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000069279	Drug Resistant Epilepsy

Ancestor Terms

ID	Term
D004827	Epilepsy
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D005690	Galactose

Ancestor Terms

ID	Term
D006601	Hexoses
D009005	Monosaccharides
D000073893	Sugars
D002241	Carbohydrates

Browse Leaves

Not provided

Browse Branches

Not provided