Trial Outcomes & Findings for Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease (NCT NCT01882062)
NCT ID: NCT01882062
Last Updated: 2026-07-31
Results Overview
The Pi/PCr Ratio is a measure of brain metabolism and it is an index of mitochondrial oxidative regulation. A 6-cm 31P transmit/receive surface coil (RAPID Biomedical GmbH, Rimpar, Germany) was used to collect free induction decays for 4 minutes at rest, 8 minutes during visual activation with 6-Hz red/black checkerboard flashes, and 8 minutes after stimulation. Subjects were able to focus on the flashes with a nonmagnetic mirror mounted above their eyes while all lights in the room were turned off. The Pi/PCr ratio was then calculated to determine brain response to cortical activation.
COMPLETED
PHASE2
10 participants
visit 1 (baseline), visit 2 (after 1 month of treatment)
2026-07-31
Participant Flow
Participant milestones
| Measure |
Triheptanoin 1g/kg/Dayfor 1 Month
Patients received Triheptanoin oil at 1g/kg/day for 1 month
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease
Baseline characteristics by cohort
| Measure |
Triheptanoin Oil at 1g/kg/Day for 1 Month
n=10 Participants
Patients received Triheptanoin oil at 1g/kg/day for 1 month
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=9 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=9 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=9 Participants
|
|
Region of Enrollment
France
|
10 Participants
n=9 Participants
|
PRIMARY outcome
Timeframe: visit 1 (baseline), visit 2 (after 1 month of treatment)The Pi/PCr Ratio is a measure of brain metabolism and it is an index of mitochondrial oxidative regulation. A 6-cm 31P transmit/receive surface coil (RAPID Biomedical GmbH, Rimpar, Germany) was used to collect free induction decays for 4 minutes at rest, 8 minutes during visual activation with 6-Hz red/black checkerboard flashes, and 8 minutes after stimulation. Subjects were able to focus on the flashes with a nonmagnetic mirror mounted above their eyes while all lights in the room were turned off. The Pi/PCr ratio was then calculated to determine brain response to cortical activation.
Outcome measures
| Measure |
Triheptanoin 1g/kg/Day for 1 Month
n=10 Participants
All patients received Triheptanoin oil at 1g/kg/day during 1 month
|
|---|---|
|
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Pi/PCr - rest at visit#1
|
0.44 ratio
Standard Deviation 0.09
|
|
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Pi/PCr - visual stimulation at visit#1
|
0.43 ratio
Standard Deviation 0.04
|
|
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Pi/PCr - recovery at visit#1
|
0.44 ratio
Standard Deviation 0.08
|
|
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Pi/PCr - rest at visit#2
|
0.42 ratio
Standard Deviation 0.05
|
|
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Pi/PCr - visual stimulation at visit#2
|
0.45 ratio
Standard Deviation 0.05
|
|
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Pi/PCr - recovery at visit#2
|
0.42 ratio
Standard Deviation 0.04
|
Adverse Events
Triheptanoin 1g/kg/Day for 1 Month
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place