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Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

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ClinicalTrials.gov Identifier: NCT02968953
Expanded Access Status : No longer available
First Posted : November 21, 2016
Last Update Posted : February 8, 2019
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Jerry Vockley, MD, PhD, University of Pittsburgh

Study Description
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Brief Summary:
This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome.

Condition or disease Intervention/treatment
Glucose Transporter 1 Deficiency Syndrome Drug: Triheptanoin

Detailed Description:
This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome. Patient has already tried the other acceptable treatments for GLUT1 with either side effects that caused treatment to be discontinued (Ketogenic Diet) or a current treatment that is not showing any efficacy and is difficult to sustain for a long duration given this patient's age (modified Atkins diet). Due to the patient's ongoing neuroregression and lack of other viable treatments, the use of UX007 (triheptanoin) is warranted. There is no comparable or satisfactory alternative therapy to treat this patient's severely debilitating condition and the potential benefits to the patient justify the potential risks of the treatment. The potential risks from UX007 (triheptanoin) are not unreasonable given the context of this patient's condition.
Study Design
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Study Type : Expanded Access
Expanded Access Type : Individual Patients
Official Title: Treatment With UX007 (Triheptanoin) for a Single Patient (ERS) With Glucose Transporter 1 (GLUT1) Deficiency Syndrome

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • Drug: Triheptanoin
    A single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome will receive tripheptanoin (UX007).
    Other Name: UX007
Eligibility Criteria
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Information from the National Library of Medicine

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

Inclusion Criteria:

  • This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome.

Exclusion Criteria:

  • Not applicable as study only includes one patient.
Contacts and Locations
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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): NCT02968953


Locations
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United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
Jerry Vockley, MD, PhD
Ultragenyx Pharmaceutical Inc
Investigators
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Principal Investigator: Gerard Vockley, MD, PhD University of Pittsburgh
More Information
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Publications:

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Responsible Party: Jerry Vockley, MD, PhD, Professor, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT02968953    
Other Study ID Numbers: PRO16060524
First Posted: November 21, 2016    Key Record Dates
Last Update Posted: February 8, 2019
Last Verified: February 2019
Additional relevant MeSH terms:
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Syndrome
Disease
Pathologic Processes