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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02599961




Registration number
NCT02599961
Ethics application status
Date submitted
3/11/2015
Date registered
9/11/2015
Date last updated
11/06/2020

Titles & IDs
Public title
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
Scientific 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
Secondary ID [1] 0 0
2015-000389-69
Secondary ID [2] 0 0
UX007G-CL202
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Glucose Transporter Type 1 Deficiency Syndrome 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - UX007

Experimental: UX007 - UX007 dosing targeted and/or maintained at 35% of total daily caloric intake.


Treatment: Drugs: UX007
UX007 is a liquid intended for oral (PO) administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths
Timepoint [1] 0 0
From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
Secondary outcome [1] 0 0
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks
Timepoint [1] 0 0
Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36
Secondary outcome [2] 0 0
Change From Baseline Over Time in CNS Total Score
Timepoint [2] 0 0
Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36
Secondary outcome [3] 0 0
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score
Timepoint [3] 0 0
Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30
Secondary outcome [4] 0 0
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score
Timepoint [4] 0 0
Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30
Secondary outcome [5] 0 0
Change From Baseline Over Time in SF-12v2 Health Survey PCS Score
Timepoint [5] 0 0
Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18
Secondary outcome [6] 0 0
Change From Baseline Over Time in SF-12v2 Health Survey MCS Score
Timepoint [6] 0 0
Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18

Eligibility
Key inclusion criteria
* Diagnosis of Glut 1 DS confirmed by cerebrospinal fluid glucose concentration. erythrocyte 3-O-methyl-D-glucose uptake assay, or solute carrier family 2 member 1 (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 (NCT01993186). 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 menses 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.
Minimum age
1 Year
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 antiepileptic drugs 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/09/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
22/10/2019
Sample size
Target
Accrual to date
Final
15
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Melbourne Brain Centre - Heidelberg
Recruitment postcode(s) [1] 0 0
3084 - Heidelberg
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
United States of America
State/province [5] 0 0
Washington
Country [6] 0 0
Denmark
State/province [6] 0 0
Copenhagen
Country [7] 0 0
Spain
State/province [7] 0 0
Barcelona
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ultragenyx Pharmaceutical Inc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.
Trial website
https://clinicaltrials.gov/study/NCT02599961
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Ultragenyx Pharmaceutical Inc
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02599961