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

Study Purpose:

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.


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling


Phase I ,Phase II

Study Chair(s)/Principal Investigator(s):

Richard S. Bedlack, MD, PhD, Duke ALS Clinic

Clinicaltrials.gov ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

Duke University
Richard S. Bedlack, MD, PhD / .(JavaScript must be enabled to view this email address) / 919-668-2839
Karen Grace, RN / .(JavaScript must be enabled to view this email address) / 919-668-2844
Durham, North Carolina 27705 United States

Full Study Summary:

Study Sponsor:

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

Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to alsconsortium.org:

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Inclusion Criteria:

    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

    Exclusion Criteria:

    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

  • Site Contact Information

    Duke University
    Durham, North Carolina 27705
    United States