A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

Completed · Phase 1/Phase 2

Conditions studied: ALS

In brief

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.

Key facts

Study ID

NCT03506425

Run by

Richard Bedlack, M.D., Ph.D.

People needed

15

Starts

2018-06-21

Expected to finish

2019-03-28

Last updated by the study team

2019-12-24

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Lab supported probable or more definite ALS by El Escorial Criteria
• Age greater than or equal to 18 years
• Willing and able to provide informed consent
• On riluzole at a stable dose for at least 30d or not taking this
• On Radicava at a stable dose for at least 30d or not taking this
• Life expectancy at least 6 months
• Currently managed on a reasonably stable diet, avoidance of fasting, carnitine or medium chain triglyceride (MCT) oils
• Must stop any other experimental ALS treatment for at least 30 days prior to screening
• If sexually active, must agree to use contraceptive or abstinence for duration of treatment with triheptanoin
• Females of child bearing age must have negative pregnancy test at screening

You may not qualify if…

• Unwilling or unable to provide informed consent
• Previous intolerance or adverse reaction to triheptanoin or MCT
• Conditions that will prohibit MRI scanning (metal in eye, some surgical implants, claustrophobia, inability to lie supine)
• Have any other co-morbid conditions that in the opinion of the study investigator, places the participant at increased risk of complications, interferes with study participation or compliance, or confounds study objectives

Where it is running

• Duke University — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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