Trial Outcomes & Findings for Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease (NCT NCT01882062)

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

visit 1 (baseline), visit 2 (after 1 month of treatment)

Results posted on

2026-03-25

Participant Flow

Participant milestones

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

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=138 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=138 Participants
Age, Categorical >=65 years	0 Participants n=138 Participants
Sex: Female, Male Female	5 Participants n=138 Participants
Sex: Female, Male Male	5 Participants n=138 Participants
Region of Enrollment France	10 Participants n=138 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

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

SECONDARY outcome

Timeframe: 1 month

Correlating an improvement of brain energy profile with clinical parameters in Huntington patients such as the Unified Huntington's disease rating scale (UHDRS) and total functional capacity score (TFC)

Outcome measures

Outcome data not reported

Adverse Events

Triheptanoin 1g/kg/Day for 1 Month

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr Fanny MOCHEL

Inserm U1127

Phone: +33 (0)1 5727 46 78

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place