Proof of Concept of an Anaplerotic Study Using Brain Phosphorus Magnetic Resonance Spectroscopy in Huntington Disease (TRIHEP2)

• ClinicalTrials.gov Identifier: NCT01882062
• Recruitment Status: Completed
• First Posted: June 20, 2013
• Results First Posted: March 24, 2016
• Last Update Posted: March 24, 2016
• Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Information provided by (Responsible Party): Institut National de la Santé Et de la Recherche Médicale, France

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Huntington Disease
• Intervention: Drug: Triheptanoin 1g/kg/day
• Enrollment: 10

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Triheptanoin Oil at 1g/kg/Day for 1 Month
Arm/Group Description	Patients received triheptanoin oil at 1g/kg/day for 1 month
Period Title: Overall Study
Started	10 [1]
Completed	10 [2]
Not Completed	0
[1] 16th of may 2013; [2] 18th of july 2013

Baseline Characteristics

Arm/Group Title		Triheptanoin Oil at 1g/kg/Day for 1 Month
Arm/Group Description		Patients received triheptanoin oil at 1g/kg/day for 1 month
Overall Number of Baseline Participants		10
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	10 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	10 100.0%
	>=65 years	0 0.0%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	10 participants
	Female	5 50.0%
	Male	5 50.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
France	Number Analyzed	10 participants
		10

Outcome Measures

1. Primary Outcome

Title	Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
Description	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.
Time Frame	visit 1 (baseline), visit 2 (after 1 month of treatment)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Triheptanoin Oil at 1g/kg/Day for 1 Month
Arm/Group Description:	All the participants received triheptanoin oil at 1g/kg/day for 1 month
Overall Number of Participants Analyzed	10
Mean (Standard Deviation); Unit of Measure: ratio
Pi/PCr - rest at visit#1	0.44 (0.09)
Pi/PCr - visual stimulation at visit#1	0.43 (0.04)
Pi/PCr - recovery at visit#1	0.44 (0.08)
Pi/PCr - rest at visit#2	0.42 (0.05)
Pi/PCr - visual stimulation at visit#2	0.45 (0.05)
Pi/PCr - recovery at visit#2	0.42 (0.04)

2. Secondary Outcome

Title	Correlation Between Primary Outcome Measure and Clinical Parameters
Description	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).
Time Frame	visit 1 (baseline), visit 2 (after 1 month of treatment)

Outcome Measure Data Not Reported

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Triheptanoin Oil at 1g/kg/Day for 1 Month
Arm/Group Description	All the participants received triheptanoin oil at 1g/kg/day for 1 month
All-Cause Mortality
	Triheptanoin Oil at 1g/kg/Day for 1 Month
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	Triheptanoin Oil at 1g/kg/Day for 1 Month
	Affected / at Risk (%)
Total	0/10 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Triheptanoin Oil at 1g/kg/Day for 1 Month
	Affected / at Risk (%)
Total	0/10 (0.00%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Dr Fanny MOCHEL
• Organization: Inserm U1127
• Phone: +33 (0)1 5727 46 78
• EMail: fanny.mochel@upmc.fr

Publications of Results:

Adanyeguh IM, Rinaldi D, Henry PG, Caillet S, Valabregue R, Durr A, Mochel F. Triheptanoin improves brain energy metabolism in patients with Huntington disease. Neurology. 2015 Feb 3;84(5):490-5. doi: 10.1212/WNL.0000000000001214. Epub 2015 Jan 7.

• Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
• ClinicalTrials.gov Identifier: NCT01882062
• Other Study ID Numbers: C12-62
• First Submitted: June 18, 2013
• First Posted: June 20, 2013
• Results First Submitted: April 30, 2015
• Results First Posted: March 24, 2016
• Last Update Posted: March 24, 2016