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Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS (GLUT-HEP)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02014883
Recruitment Status : Unknown
Verified January 2014 by Institut National de la Santé Et de la Recherche Médicale, France.
Recruitment status was:  Recruiting
First Posted : December 18, 2013
Last Update Posted : May 1, 2015
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

Brief Summary:
The purpose of this project is to study the efficacy of triheptanoin oil in patients with GLUT1 deficiency syndrome.

Condition or disease Intervention/treatment Phase
Glut1 Deficiency Syndrome Drug: GLUT1 DS Phase 2

Detailed Description:

The primary objective of the study is:

- to evaluate the capacity of triheptanoïn to improve the condition of patients with GLUT1-DS

The secondary objectives of the study are:

  • to confirm the short-term safety of triheptanoïn therapy in patients with GLUT1-DS
  • to evaluate the short-term effects of triheptanoïn treatment on motor function, autonomy, quality of life and clinical signs of patients with GLUT1-DS
  • to evaluate the effect of triheptanoïn on brain energy metabolism using non-invasive 31P-MRS spectroscopy after activation of the occipital cortex in order to measure the levels of high-energy phosphates (such as ATP and phosphocreatine)

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS
Study Start Date : December 2013
Estimated Primary Completion Date : May 2015
Estimated Study Completion Date : April 2018


Arm Intervention/treatment
Experimental: GLUT1 DS Drug: GLUT1 DS



Primary Outcome Measures :
  1. Number of paroxystic events [ Time Frame: 6 months ]
    The number of paroxystic events, in particular abnormal movements, will be collected during trihepatnoin treatment.


Secondary Outcome Measures :
  1. Safety [ Time Frame: 6 months ]
    Should the whole blood levels of propionylcarnitine increase above 8 μmol/l, the dose of triheptanoin will be reduced until the decrease of whole blood propionylcarnitine is below 8 μmol/l. Should an organic acid abnormality such as an excessive urinary excretion of propionate metabolites such as 3-hydroxypropionic, 2-methylcitric, propionylglycine, tiglylglycine and/or methylmalonic acid occur, the dose of triheptanoin will be reduced until normalization of the organic acid and acylcarnitine profile. If still abnormal, patient will be excluded from the study. For GI distress, the research dietitian will instruct the patient regarding taking the dose over a longer period of time (30 minutes). If GI distress persists, triheptanoin dose will be reduced by 50% and re-increased progressively as the problems resolve with the patients working closely with research dietitian until tolerance of the full dose is achieved.

  2. 6 minutes walk test [ Time Frame: 6 months ]
  3. 9 hole Peg board [ Time Frame: 6 months ]
  4. Clinical Global Impression Scales [ Time Frame: 6 months ]
  5. Schwab-England scale [ Time Frame: 6 months ]
  6. Vineland Scale [ Time Frame: 6 months ]
  7. Fatigue Severity Scale [ Time Frame: 6 months ]
  8. Fatigue Visual Scale [ Time Frame: 6 months ]
  9. Brain 31phosphorus magnetic resonance spectroscopy [ Time Frame: 6 months ]
    Ratio of Inorganic Phosphate (Pi) over Phosphocreatine during visual stimulation



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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02014883


Contacts
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Contact: Marie-Pierre Luton, PhD +33 1 57 27 44 82 mariepierre.luton@icm-institute.org

Locations
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France
Brain and Spine Institute Recruiting
Paris, France, 75013
Sub-Investigator: Elodie Hainque, MD         
Sub-Investigator: Domitille Gras, MD         
Sponsors and Collaborators
Institut National de la Santé Et de la Recherche Médicale, France
Ultragenyx Pharmaceutical Inc
Investigators
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Principal Investigator: Fanny Mochel, MD, PhD Institut National de la Santé Et de la Recherche Médicale, France
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT02014883    
Other Study ID Numbers: C13-37
2013-A01300-45 ( Registry Identifier: IDRCB )
First Posted: December 18, 2013    Key Record Dates
Last Update Posted: May 1, 2015
Last Verified: January 2014