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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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7 September 2021 |
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Main ID: |
NCT02014883 |
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Date of registration:
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03/12/2013 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS
GLUT-HEP |
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Scientific title:
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Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS |
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Date of first enrolment:
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December 4, 2013 |
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Target sample size:
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20 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02014883 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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France
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Contacts
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Name:
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Fanny Mochel, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Institut National de la Santé Et de la Recherche Médicale, France |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Mutation in SLC2A1 gene
- Age > 3 years
- Patient with history/frequency of seizures or movement disorders documented at least 3
months prior to the beginning of the study
- Covered by french social security
- Patients who freely agree to participate in this study and understand the nature,
risks and benefits of this study and give their written informed consent. (In addition
to the requirement for the consent of parents or the legal representative, adolescents
can provide additional informed consent to participate in clinical trials)
Exclusion Criteria:
- Evidence of psychiatric disorder
- Attendant neurological disorder
- Comorbid medical condition that would render them unsuitable for the study, e.g. HIV,
diabetes
- Pregnant or parturient or lactating women
- Unwillingness to be informed in case of abnormal MRI
- Failure to give written informed consent
- Unable to understand the protocol
- Unable to participate to the whole study
- Absence of signed informed consent
- Persons deprived of their liberty by judicial or administrative decision
- Person subject to an exclusion period for another research
- Subjects with exclusion criteria required by french law
Age minimum:
3 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Glut1 Deficiency Syndrome
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Intervention(s)
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Drug: GLUT1 DS
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Primary Outcome(s)
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Number of paroxystic events
[Time Frame: 6 months]
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Secondary Outcome(s)
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9 hole Peg board
[Time Frame: 6 months]
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Fatigue Visual Scale
[Time Frame: 6 months]
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Brain 31phosphorus magnetic resonance spectroscopy
[Time Frame: 6 months]
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Safety
[Time Frame: 6 months]
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6 minutes walk test
[Time Frame: 6 months]
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Vineland Scale
[Time Frame: 6 months]
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Fatigue Severity Scale
[Time Frame: 6 months]
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Schwab-England scale
[Time Frame: 6 months]
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Clinical Global Impression Scales
[Time Frame: 6 months]
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Secondary ID(s)
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C13-37
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2013-A01300-45
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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