A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
Status:
Completed
Trial end date:
2019-03-28
Target enrollment:
Participant gender:
Summary
The causes of ALS are largely unknown. However, mitochondrial dysfunction, resulting in
impaired energy production, oxidative stress and apoptosis, may play a key role in ALS
progression. Triheptanoin can improve mitochondrial function and energy production and
therefore has potential for slowing ALS progression. Indeed, triheptanoin slowed motor neuron
loss and delayed the onset of weakness in a mutant SOD1 model of ALS. This pilot trial will
determine if Triheptanoin is safe tolerable, alters biomarkers of brain energy metabolism and
oxidative stress, and slows functional decline in people with ALS.