Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

NCT02599961 | Terminated Phase 2 Results

• 2 other identifiers: UX007G-CL2022015-000389-69
• Study Type: interventional
• Target: 15
• Locations: 5 countries
• Sites: 9

Brief Summary

The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.

Conditions

Glucose Transporter Type 1 Deficiency Syndrome

Keywords

Glut 1 DS

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths

  An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE.

  From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.

Secondary Outcomes (6)

• Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks

  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

• Change From Baseline Over Time in CNS Total Score

  Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36

• Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score

  Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30

• Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score

  Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30

• Change From Baseline Over Time in SF-12v2 Health Survey PCS Score

  Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18

Study Arms (1)

UX007

EXPERIMENTAL

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

Drug: UX007

Interventions

UX007 DRUG

UX007 is a liquid intended for oral (PO) administration.

Also known as: Triheptanoin

UX007

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Ultragenyx Pharmaceutical Inc: lead

Study Sites (9)

Children's Hospital Colorado - University of Colorado, Denver, School of Medicine

Aurora, Colorado, 80045, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Melbourne Brain Centre

Heidelberg, Victoria, 3084, Australia

Location

Copenhagen University Hospital

Copenhagen, 2100, Denmark

Location

Hospital Sant Joan De Deu

Barcelona, 08950, Spain

Location

The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, NE7 7DN, United Kingdom

Location

Related Links

• Company Website

MeSH Terms

Conditions

Glut1 Deficiency Syndrome

Interventions

triheptanoin

Results Point of Contact

• Title: Medical Information
• Organization: Ultragenyx Pharmaceutical Inc

medinfo@ultragenyx.com

1-888-756-8657

Study Officials

• Medical Director

  Ultragenyx Pharmaceutical Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: November 3, 2015
• First Posted: November 9, 2015
• Study Start: September 10, 2015
• Primary Completion: October 22, 2019
• Study Completion: October 22, 2019
• Last Updated: June 11, 2020
• Results First Posted: April 30, 2020

Record last verified: 2020-06

Locations

US (5); DK (1); AU (1); ES (1); GB (1)