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Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

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: 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

Tracking Information
First Submitted Date November 16, 2016
First Posted Date November 21, 2016
Last Update Posted Date February 8, 2019
 
Descriptive Information
Brief Title Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome
Official Title Treatment With UX007 (Triheptanoin) for a Single Patient (ERS) With Glucose Transporter 1 (GLUT1) Deficiency Syndrome
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.
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 Type Expanded Access
Expanded Access Type Individual Patients
Condition Glucose Transporter 1 Deficiency Syndrome
Intervention Drug: Triheptanoin
A single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome will receive tripheptanoin (UX007).
Other Name: UX007
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Expanded Access Status No longer available
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: Female
Ages 3 Years and older   (Child, Adult, Older Adult)
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02968953
Other Study ID Numbers PRO16060524
Current Responsible Party Jerry Vockley, MD, PhD, University of Pittsburgh
Original Responsible Party Amy Goldstein, University of Pittsburgh, Assistant Professor
Current Study Sponsor Jerry Vockley, MD, PhD
Original Study Sponsor Amy Goldstein
Collaborators Ultragenyx Pharmaceutical Inc
Investigators
Principal Investigator: Gerard Vockley, MD, PhD University of Pittsburgh
PRS Account University of Pittsburgh
Verification Date February 2019