TRIHEP3- At a Glance
  • Sponsored by : Institut National de la Santé Et de la Recherche Médicale, France
  • Collaborator: Ultragenyx Pharmaceutical Inc
  • Official Title: A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington’s Disease
  • Involves: Triheptanoin oil orally administered at 1g/kg/day
  • Study type: Clinical Interventional trial
  • phase: Not applicable 
  • Purpose : 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 and brain energy metabolism as evaluated by the ratio of inorganic phosphate/phosphocreatine, during visual stimulation, using 31P-MRS. Clinical improvement will be evaluated by UHDRS, TFC, and PBA-S scores as well as performance on the neuropsychological battery; patient quality of life will be evaluated with qualitative research methods after 6 months and 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.
  • Status: see trial sites below for details on recruitment status

 

Eligibility Criteria

 

Ages Eligible for Study:  18 Years and older  (Adult, Older Adult)
Sexes Eligible for Study:  All
Accepts Healthy Volunteers: No

 

Inclusion Criteria

  • Positive genetic test with CAG repeat length ≥39 in HTT gene
  • At least 18 years of age
  • Signature of informed consent
  • Covered by social security
  • UHDRS score between 5 and 40
  • Ability to undergo MRI scanning
  • BMI between 18 and 30

Exclusion Criteria

  • Hypersensitivity to resveratrol or to one of its excipients (gelatin and glycerin)
  • Tetrabenazine treatment
  • Neuroleptic treatments other than olanzapine at small doses (≤10 mg) and Abilify® (≤15mg)
  • VKA treatment (Previscan®, Sintron®, Coumadine®)
  • NACO treatment (Pradaxa®, Xarelto®, Eliquis®)
  • Additional psychiatric or neurological conditions
  • Severe head injury
  • Participation in another therapeutic trial (3 months exclusion period)
  • Pregnancy and breastfeeding
  • Inability to understand information about the protocol
  • Persons deprived of their liberty by judicial or administrative decision
  • Adult subject under legal protection or unable to consent.

 

TRIAL SITES

 

FRANCE

 

Trial site:  Département de Génétique

 

Status: Active, recruitment completed 

Address: Paris, France, 75013

 

 

 

Contact: Not Available at the moment

 

Telephone: Not Available at the moment

 

Email:  Not Available at the moment

 

 

 

NETHERLANDS

 

Trial site: Department of Neurology

 

Status: Active, recruitment completed 

Address: Leiden, Netherlands, 2300RC

 

 

Contact: Not Available at the moment

 

Telephone: Not Available at the moment

 

Email:  Not Available at the moment