Working…
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study to Assess the Long Term Safety and Efficacy of UX007 in Subjects With Glucose Type 1 DS

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: NCT02599961
Recruitment Status : Terminated (Study was halted prematurely due to lack of efficacy)
First Posted : November 9, 2015
Last Update Posted : November 1, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
UX007G-CL202 is an open-label, single-arm, multi center extension study to assess the long-term safety and efficacy of UX007 in Glut 1 DS. The study will enroll up to 40 pediatric, adolescent and adult Glut 1 DS subjects who have completed the UX007G-CL201 study or participated in qualified ISTs.

Condition or disease Intervention/treatment Phase
Glucose Transporter Type 1 Deficiency Syndrome Drug: UX007 Phase 2

Detailed Description:

A Phase 2 study (UX007G-CL-201) is currently ongoing to assess safety and efficacy of UX007 in reducing the frequency of seizures in Glut 1 DS patients. ISTs and other treatment programs are also evaluating UX007 as a treatment for Glut 1 DS.

This open label long term safety and efficacy study (UX007G-CL202) will provide an opportunity for Glut 1 DS patients to be treated with UX007 for up to 3 years (or until market approval) under a single standardized protocol for maintenance therapy and consistent safety monitoring. The study is designed to obtain long-term safety information and evaluate maintenance of efficacy in a pediatric and adult Glut 1 DS population.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome
Actual Study Start Date : September 2015
Actual Primary Completion Date : October 22, 2019
Actual Study Completion Date : October 22, 2019


Arm Intervention/treatment
Experimental: UX007 (Triheptanoin)
This is a single arm, open label study. All subjects will be on active treatment; no reference therapy or placebo will be administered.
Drug: UX007
UX007 is a liquid intended for oral (PO) administration.
Other Name: Triheptanoin




Primary Outcome Measures :
  1. Frequency of AEs and SAEs [ Time Frame: 3 years ]
    Frequency of Adverse Events and Serious Adverse Events assessed as related to study drug


Secondary Outcome Measures :
  1. Maintenance of reduction in seizure frequency [ Time Frame: 3 years ]
  2. Long term Efficacy of UX007 [ Time Frame: 3 years ]
    Long term efficacy in measures of neurological function and development delay using the Cambridge Neuropsychological Test Automated Battery (CANTAB)

  3. Long term Efficacy of UX007 [ Time Frame: 3 years ]
    Long term efficacy in measures of neurological function and development delay using the Columbia Neurological Score

  4. Maintenance of UX007 treatment effect on gross motor function and movement disorders using the 6 Minute Walk Test [ Time Frame: 3 years ]
  5. Maintenance of UX007 treatment effect on gross motor function and movement disorders using Paroxymal Exertional Dyskinesia (PED) [ Time Frame: 3 years ]
  6. Continued impact of UX007 treatment on clinical status and health related quality of life using the Clinical Global Impression Scales [ Time Frame: 3 years ]
  7. Continued impact of UX007 treatment on clinical status and health related quality of life using the Short Form Health Surveys [ Time Frame: 3 years ]
  8. Continued impact of UX007 treatment on clinical status and health related quality of life using thePediatric Evaluations of Disability Inventory Computer Adaptive Test (PEDI-CAT) [ Time Frame: 3 years ]


Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of Glut 1 DS confirmed by cerebrospinal fluid glucose concentration. erythrocyte 3-O-methyl-D-glucose uptake assay, or SLC2A1 molecular genetic testing (Information obtained from Medical Records)
  • Males and females aged at least 1 year old at the time of informed consent
  • Completion of UX007G-CL201 study (NCT - 01993186). Glut1 DS patients who received UX007/triheptanoin treatment as apart of clinical studies, ISTs or expanded access/compassionate use treatment programs may be eligible at the discretion of the Sponsor
  • Provide written informed consent or verbal assent (if possible) with written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research related procedures
  • Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, and comply with accurate completion of the seizure diary
  • Females of childbearing potential must have a negative urine pregnancy test at Baseline and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, are post-menopausal (defined as having no mensus for at least 12 months without an alternative medical cause), or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
  • Participants of child-bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly-effective method of contraception as determined by the investigator from the period following the signing of the informed consent through 30 days after last dose of study drug.

Exclusion Criteria:

  • Any known hypersensitivity to triheptanoin, that in the judgement of the investigator, places the subject at an increased risk for adverse effects
  • History of, or current suicidal ideation, behavior and/or attempts
  • Pregnant and/or breast feeding an infant
  • Unwilling or unable to discontinue use of prohibited medication (barbiturates, pancreatic lipase inhibitors) or other substance that may confound study objectives. Use of up to 3 concomitant AEDs is allowed, provided dose has been stable at least 14 days prior to Baseline
  • Use of any Investigational Product, drug or supplement (other than UX007) within 30 days prior to Baseline, or at any time during the study
  • Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment
  • Has a concurrent disease or condition, or laboratory abnormality that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduce additional safety concerns

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): NCT02599961


Locations
Layout table for location information
United States, Colorado
Children's Hospital Colorado - University of Colorado, Denver, School of Medicine
Aurora, Colorado, United States, 80045
United States, Florida
Miami Children's Hospital
Miami, Florida, United States, 33155
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
United States, Texas
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Australia, Victoria
Melbourne Brain Centre
Heidelberg, Victoria, Australia, 3084
Denmark
Copenhagen University Hospital
Copenhagen, Denmark, 2100
Spain
Hospital Sant Joan De Deu
Barcelona, Spain, 08950
United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE7 7DN
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
Layout table for investigator information
Study Director: Medical Director Ultragenyx Pharmaceutical Inc

Additional Information:
Layout table for additonal information
Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT02599961    
Other Study ID Numbers: UX007G-CL202
2015-000389-69 ( EudraCT Number )
First Posted: November 9, 2015    Key Record Dates
Last Update Posted: November 1, 2019
Last Verified: October 2019
Keywords provided by Ultragenyx Pharmaceutical Inc:
Glut 1 DS
Additional relevant MeSH terms:
Layout table for MeSH terms
Carbohydrate Metabolism, Inborn Errors
Syndrome
Disease
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases