An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 8 February 2021
• Main ID: NCT02036853
• Date of registration: 13/01/2014
• Prospective Registration: Yes
• Primary sponsor: Adrian Lacy
• Public title: An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome GLUT1DS
• Scientific title: An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome (GLUT1 DS)
• Date of first enrolment: February 20, 2014
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02036853
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Adrian Lacy, MD
• Affiliation: Cook Children's Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

Individuals eligible to participate in this study must meet all of the following criteria:

1. Patients with GLUT1 DS by physician diagnosis

2. Males and females, aged 1 to 50 years

3. Allowed to be on concomitant AEDs

4. Patients are able to tolerate triheptanoin if they have been (or are currently being)
treated with this medication

5. Must, in the opinion of the investigator, be willing and able to comply with study
procedures and schedule

6. Provide written assent (if appropriate) and written informed consent by a Legally
Authorized Representative (LAR) after the nature of the study has been explained, and
prior to any research-related procedures

7. Sexually active subjects must be willing to use an acceptable method of contraception
while participating in the study

8. Females of childbearing potential must have a negative pregnancy test at Screening and
be willing to have additional pregnancy tests during the study

Exclusion Criteria:

Individuals who meet any of the following exclusion criteria will not be eligible to
participate in the study:

1. Patients and their Legally Authorized Representatives (as appropriate) not willing or
able to give written or verbal assent or written informed consent.

2. Concomitant administration of a ketogenic diet for the treatment of GLUT1 deficiency

3. Concomitant administration of valproic acid

4. In the Investigator's opinion, the patient may not be compliant

5. Pregnant or breastfeeding an infant at screening

6. Has a concurrent disease or condition, or laboratory abnormality that, in the view of
the Investigator, places the subject at high risk for adverse events, or introduces
additional safety concerns

7. History of or current suicidal ideation, behavior and attempts

8. Patient qualifies for any other clinical trial designed to progressively evaluate the
safety and efficacy of triheptanoin as approved by the FDA under a separate IND which
is open at Cook Children's



Age minimum: 1 Year
Age maximum: 50 Years
Gender: All

Health Condition(s) or Problem(s) studied

Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

Intervention(s)

Drug: Triheptanoin

Primary Outcome(s)

Reported Change in Seizures Frequency From Baseline at 13 Weeks [Time Frame: Baseline and 13 weeks]

Reported Change in Seizures Frequency From Baseline at 2 Years [Time Frame: Baseline and two yrs]

Reported Change in Seizures Frequency From Baseline at 3 Years [Time Frame: Baseline and three yrs]

Reported Change in Seizure Frequency From Baseline at 4 Years [Time Frame: Baseline and four yrs]

Reported Change in Seizures Frequency From Baseline at 1 Year [Time Frame: Baseline and one yr]

Reported Change in Seizure Frequency From Baseline at 5 Years [Time Frame: Baseline and five yrs]

Reported Change in Seizures Frequency From Baseline at 18 Months [Time Frame: Baseline and 18 months]

Reported Change in Seizures Frequency From Baseline at 26 Weeks [Time Frame: Baseline and 26 weeks]

Secondary ID(s)

2013-NEUR-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ultragenyx Pharmaceutical Inc

Results

• Results available: Yes
• Date Posted: 28/01/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02036853