Trial Outcomes & Findings for Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D) (NCT NCT02018315)

NCT ID: NCT02018315

Last Updated: 2019-10-31

Results Overview

Visual analysis of EEG recording to determine the fraction of spike-range within the area of recording.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 14 participants
• Primary outcome timeframe: 1 day
• Results posted on: 2019-10-31

Participant Flow

Participant milestones

Measure	Experimental: Triheptanoin Triheptanoin (C7 oil, liquid) dosed at 1 g/kg body weight divided and administered 4 times per day via mouth or g-tube for 3 months.
Overall Study STARTED	14
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)

Baseline characteristics by cohort

Measure	Experimental: Triheptanoin n=14 Participants Triheptanoin (C7 oil, liquid) dosed at 1 g/kg body weight divided and administered 4 times per day via mouth or g-tube for 3 months.
Age, Categorical <=18 years	11 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	12.5 Years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	14 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 day

Visual analysis of EEG recording to determine the fraction of spike-range within the area of recording.

Outcome measures

Measure	Experimental: Triheptanoin n=14 Participants Triheptanoin (C7 oil, liquid) dosed at 1 g/kg body weight divided and administered 4 times per day via mouth or g-tube for 3 months.
Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time	13 Participants

SECONDARY outcome

Timeframe: 3 months

Population: 5 participants out of the 14 total participants enrolled completed the optional MRI . Reasons for participants electing to not participate in imaging included inability to remain immobile due to movement disorder or anxiety, immaturity, metal implants, and personal choice.

Magnetic Resonance Imaging (MRI) used to calculate brain metabolic rate. Brain metabolic rate compared before oil ingestion (Baseline), 90 minutes after oil ingestion, and after 3 months of daily oil ingestion in each participant. Triheptanoin metabolism may lead to increased oxygen consumption only while the brain undergoes a reduction of ictogenesis. We hypothesize that when ictogenesis is abolished by triheptanoin or absent at baseline, triheptanoin exerts little or no effect on CMR02.

Outcome measures

Measure	Experimental: Triheptanoin n=5 Participants Triheptanoin (C7 oil, liquid) dosed at 1 g/kg body weight divided and administered 4 times per day via mouth or g-tube for 3 months.
Number of Participants With Change in Brain Metabolic Rate After 3 Months	5 Participants

Adverse Events

Triheptanoin

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Triheptanoin n=14 participants at risk Triheptanoin (C7 oil, liquid) dosed at 1 g/kg body weight divided and administered 4 times per day via mouth or g-tube for 3 months. Triheptanoin: Triheptanoin is a 7-carbon medium chain triglyceride
Gastrointestinal disorders Gastric discomfort	7.1% 1/14
Metabolism and nutrition disorders weight gain	7.1% 1/14
Gastrointestinal disorders Diarrhea	14.3% 2/14

Additional Information

Juan Pascual, M.D., Ph.D.

University of Texas Southwestern Medical Center

Phone: 214-648-3550

Email: juan.pascual@utsouthwestern.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place