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Post Study Continuation of C7 for G1D

Expanded access is currently available for this treatment.
Verified December 2013 by University of Texas Southwestern Medical Center
Sponsor:
Information provided by (Responsible Party):
Juan Pascual, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier:
NCT02018302
First received: December 11, 2013
Last updated: December 26, 2013
Last verified: December 2013
  Purpose

This protocol provides ongoing access to triheptanoin for patients who did complete an initial pilot protocol, and provides the opportunity to collect long-term safety data from patients treated with triheptanoin.


Condition Intervention Phase
Glut1 Deficiency Syndrome
Glucose Transporter Type 1 Deficiency Syndrome
Drug: Triheptanoin
Phase 1

Study Type: Expanded Access     What is Expanded Access?
Official Title: Post Study Continuation of C7 for G1D

Resource links provided by NLM:


Further study details as provided by University of Texas Southwestern Medical Center:

Study Start Date: January 2013
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Triheptanoin
    Other Names:
    • C7 oil
    • Heptanoate
    • heptanoic acid
Detailed Description:

Only patients who completed the initial pilot study were eligible. The study is ongoing with these patients only.

  Eligibility

Ages Eligible for Study:   1 Month to 28 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients enrolled in the pilot trial (UTSW 122010-186)

Exclusion Criteria:

  • Subjects who did not complete the pilot trial (UTSW 122010-186)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02018302

Contacts
Contact: Dorothy Kelly, MA rare.diseases@utsouthwestern.edu

Locations
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Juan Pascual
Investigators
Principal Investigator: Juan M Pascual, MD, PhD UT Southwestern Medical Center
  More Information

Additional Information:
Publications:

Responsible Party: Juan Pascual, Associate Professor, Director of the Rare Brain Disorders Program, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT02018302     History of Changes
Other Study ID Numbers: UTSW 082013-016
Study First Received: December 11, 2013
Last Updated: December 26, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Texas Southwestern Medical Center:
G1D
Glut1 Deficiency
Glucose Transporter Type 1 Deficiency
Glucose Transporter Type I Deficiency

Additional relevant MeSH terms:
Carbohydrate Metabolism, Inborn Errors
Syndrome
Disease
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Pathologic Processes

ClinicalTrials.gov processed this record on November 25, 2014