Clinical Long Term Evaluation of Glutamine Supplement in... | NCT05255328

Studies
Clinical Long Term Evaluation of Glutamine Supplement in MELAS Syndrome

NCT05255328

Sponsor

Hospital Universitario 12 de Octubre

Status

Unknown status

Last Update Posted

Sep 28, 2023Actual

Enrollment

9Actual

Phase

Not Applicable

Conditions

MELAS Syndrome

Interventions

Glutamine oral supplement

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

Sep 2023

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Active, not recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 1, 2021Actual

Primary Completion Date

Jul 15, 2022Actual

Completion Date

Jul 24, 2024Estimated

First Submitted Date

Jan 19, 2022

First Submission Date that Met QC Criteria

Feb 23, 2022

First Posted Date

Feb 24, 2022Actual

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

Sep 27, 2023

Last Update Posted Date

Sep 28, 2023Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Jesús González de la Aleja Tejera, Jesús González de la Aleja Tejera, MD, PhD, Principal Investigator, Hospital Universitario 12 de OctubrePrincipal Investigator

Lead Sponsor

Hospital Universitario 12 de OctubreOTHER

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

The purpose of this study is to assesses the clinical efficacy of oral supplementation with glutamine over 3 years.

Detailed Description

Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) syndrome is a genetically heterogeneous disorder. The most common mutation is in the mtDNA gene MT-TL1 encoding the mitochondrial tRNALeu (UUR). For understanding the development of seizures in patients with mitochondrial disease, a study has recently emphasized the deficiency of astrocytic glutamine synthetase, creating a disinhibited neuronal network for seizure generation. The investigators propose to evaluate nine patients with mitochondrial DNA mutation and MELAS. Patients will receive oral supplementation with 12-18 g/day of glutamine (adjusted for weight and plasma concentrations). The primary outcome measures modification in clinical scales.

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

9Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
MELAS	Experimental	Patients with MELAS syndrome will receive 12-18g/day of glutamine	Dietary Supplement: Glutamine oral supplement

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Glutamine oral supplement	Dietary Supplement	12-18 g /day of glutamine supplementation	MELAS

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Clinical efficacy; JMDRS	Change from Baseline clinical scale (Japanese mitochondrial disease rating scale (JMDRS)) at 12,24,36 months to test Clinical efficacy of oral supplementation	36 months
Clinical efficacy; MMSE	Change from Baseline cognitive test (Mini-Mental State Examination (MMSE)) at 12,24,36 months to test Clinical efficacy of oral supplementation	36 months

Secondary Outcomes

Measure	Description	Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0	To recorde all adverse events grades; with special interest on investigations (CTCAE v5.0, 2017)	36 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

MELAS syndrome Clinically and genetically confirmed.
Patients have already participated in GLN-9-MIT study

Exclusion Criteria:

subjects harboring a MELAS-related pathogenic mtDNA mutation no fulfilling the complete diagnostic criteria for the MELAS phenotype

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Jesús González de la Ajeja Tejera, MD, PhD	Hospital Universitario 12 Octubre	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Universitario 12 de Octubre		Madrid		28041	Spain

Condition Browse Module

MeSH Terms

ID	Term
D017241	MELAS Syndrome

Ancestor Terms

ID	Term
D017237	Mitochondrial Encephalomyopathies
D017240	Mitochondrial Myopathies
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

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Browse Branches

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