MELAS SyndromeNCT05255328
Clinical Long Term Evaluation of Glutamine Supplement in MELAS Syndrome
A study testing Glutamine oral supplement in people with MELAS Syndrome, run by Hospital Universitario 12 de Octubre at 1 site in Spain.
Trial summary
The purpose of this study is to assesses the clinical efficacy of oral supplementation with glutamine over 3 years. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- MELAS Syndrome
- What is being tested?
- Glutamine oral supplement
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteSpain
- Study dates
- 1 Jul 2021to 24 Jul 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- MELAS syndrome Clinically and genetically confirmed.
- Patients have already participated in GLN-9-MIT study
Who cannot join
- subjects harboring a MELAS-related pathogenic mtDNA mutation no fulfilling the complete diagnostic criteria for the MELAS phenotype
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Long Term Evaluation of Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) Syndrome in Order to Prevent Neurological Damage.
The purpose of this study is to assesses the clinical efficacy of oral supplementation with glutamine over 3 years.
Read the 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.
- Conditions
- MELAS Syndrome
- Being tested
- Glutamine oral supplement
- Keywords
- MELAS, Glutamine, Oral supplements, Amino acids
- Medical subject headings
- MELAS Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Glutamine oral supplementDietary supplement
12-18 g /day of glutamine supplementation
| Study group | Role | What they receive |
|---|---|---|
| MELAS | Experimental | Patients with MELAS syndrome will receive 12-18g/day of glutamine Receives: Dietary Supplement: Glutamine oral supplement |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 36 months
Secondary outcomes (1)
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)
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2022
- Study start1 Jul 2021
- Primary completion15 Jul 2022
- Study completion24 Jul 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MELAS SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Glutamine oral supplementation
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital Universitario 12 de Octubre
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jesús González de la Aleja Tejera, Hospital Universitario 12 de Octubre
- Investigators
- Jesús González de la Ajeja Tejera, MD, PhD, Principal investigator, Hospital Universitario 12 Octubre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05255328
- Sponsor's protocol ID
- GLN-9-MIT2
- Status last verified
- Sep 2023
- First submitted
- 19 Jan 2022
- Last updated
- 28 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Long Term Evaluation of Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) Syndrome in Order to Prevent Neurological Damage.
- ClinicalTrials.gov ID
- NCT05255328
- Acronym / sponsor ID
- GLN-9-MIT2
- Conditions
- MELAS Syndrome
- Keywords
- MELAS, Glutamine, Oral supplements, Amino acids
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Hospital Universitario 12 de OctubreUniversity, hospital or other
- Study start
- 1 Jul 2021 (actual)
- Primary completion
- 15 Jul 2022 (actual)
- Study completion
- 24 Jul 2024 (estimated)
- First posted
- 24 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Glutamine oral supplementation
| Arm | Type | Receives |
|---|---|---|
| MELAS | Experimental | Dietary Supplement: Glutamine oral supplementPatients with MELAS syndrome will receive 12-18g/day of glutamine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- MELAS syndrome Clinically and genetically confirmed.
- Patients have already participated in GLN-9-MIT study
Key exclusion criteria (1 listed)
- subjects harboring a MELAS-related pathogenic mtDNA mutation no fulfilling the complete diagnostic criteria for the MELAS phenotype
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Glutamine oral supplement12-18 g /day of glutamine supplementation | Dietary supplement | MELAS |
Primary outcome
Clinical efficacy; JMDRS
Time frame: 36 months
Change from Baseline clinical scale (Japanese mitochondrial disease rating scale (JMDRS)) at 12,24,36 months to test Clinical efficacy of oral supplementation
Clinical efficacy; MMSE
Time frame: 36 months
Change from Baseline cognitive test (Mini-Mental State Examination (MMSE)) at 12,24,36 months to test Clinical efficacy of oral supplementation
1 secondary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Time frame: 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2023.
Related trials for MELAS Syndrome
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Not yet recruitingPhase 1/2
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
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Global Registry and Natural History Study for Mitochondrial Disorders
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North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Mitochondrial Disorders, Mitochondrial Genetic Disorders
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Natural History Study - Mitochondrial Disease
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jesús González de la Ajeja Tejera, MD, PhD principal investigator
Hospital Universitario 12 Octubre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Glutamine oral supplement in people with MELAS Syndrome, run by Hospital Universitario 12 de Octubre at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical efficacy; JMDRS (time frame: 36 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2023.