NCT02453061: TRIHEP3

A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease

Completed Phase 2 Last updated 15 July 2024

What this trial tests

Phase 2 trial testing Triheptanoin oil in Huntington Disease in 100 participants. Completed in 3 December 2019.

Timeline

29 June 2015

Primary endpoint
3 December 2019

3 December 2019

Quick facts

Lead sponsor	Institut National de la Santé Et de la Recherche Médicale, France
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	100
Start date	29 June 2015
Primary completion	3 December 2019
Estimated completion	3 December 2019
Sites	2 locations across France, Netherlands

Drugs / interventions tested

• Triheptanoin oil — full drug profile →
• Placebo

Conditions studied

• Huntington Disease — all drugs for Huntington Disease →

Sponsor

Institut National de la Santé Et de la Recherche Médicale, France — full company profile →

Who can join

18 and older, any sex, with Huntington Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

In the study the investigators plan to include 100 early affected HD patients (5 ≤ UHDRS ≤ 40) in a randomized, double-blind, controlled study in 2 centers (France and the Netherlands). Patients will receive either triheptanoin at 1g/kg of body weight per day (n = 50), or a control oil (n = 50) at 1g/kg of body weight per day for 6 months followed by an open label phase using triheptanoin for another 6 months. Efficacy of triheptanoin will be evaluated by measurements of caudate volume using volumetric magnetic resonance imaging. Clinical improvement will be evaluated by brain energy metabolism quantification as evaluated by the ratio of inorganic phosphate/phosphocreatine, during visual stimulation, using 31P-MRS, UHDRS, TFC, and PBA-S scores as well as performance on the neuropsychological battery. Patient quality of life will be evaluated with the SF-36 questionnaire before and after treatment; biological tolerance and compliance will be evaluated by routine biochemical blood tests, plasma and urine measurements of triheptanoin oil derivatives and patient report.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. New Avenues for the Treatment of Huntington's Disease.
   Kim A, Lalonde K, Truesdell A, Gomes Welter P, et al · · 2021 · cited 130× · PMID 34445070 · DOI 10.3390/ijms22168363
2. Current Pharmacological Approaches to Reduce Chorea in Huntington's Disease.
   Coppen EM, Roos RA. · · 2017 · cited 90× · PMID 27988871 · DOI 10.1007/s40265-016-0670-4
3. From Pathogenesis to Therapeutics: A Review of 150 Years of Huntington's Disease Research.
   Jiang A, Handley RR, Lehnert K, Snell RG. · · 2023 · cited 88× · PMID 37629202 · DOI 10.3390/ijms241613021
4. Therapeutic Advances for Huntington's Disease.
   Kumar A, Kumar V, Singh K, Kumar S, et al · · 2020 · cited 66× · PMID 31940909 · DOI 10.3390/brainsci10010043
5. Huntington’s Disease Clinical Trials Corner: February 2018
   Rodrigues FB, Wild EJ. · · 2018 · cited 45× · PMID 29480210 · DOI 10.3233/jhd-189001
6. Therapeutic Update on Huntington's Disease: Symptomatic Treatments and Emerging Disease-Modifying Therapies.
   Dash D, Mestre TA. · · 2020 · cited 35× · PMID 32705582 · DOI 10.1007/s13311-020-00891-w
7. Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia: An MRI Study of 16 French Cases.
   Codjia P, Ayrignac X, Mochel F, Mouzat K, et al · · 2018 · cited 35× · PMID 30115677 · DOI 10.3174/ajnr.a5744
8. Clinical Trials Corner: September 2017.
   Rodrigues FB, Wild EJ. · · 2017 · cited 33× · PMID 28968245 · DOI 10.3233/jhd-170262