A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
Status:
Completed
Trial end date:
2019-12-03
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Institut National de la Santé Et de la Recherche Médicale, France