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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03506425
• Date of registration: 13/04/2018
• Prospective Registration: Yes
• Primary sponsor: Richard Bedlack, M.D., Ph.D.
• Public title: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
• Scientific title: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
• Date of first enrolment: June 21, 2018
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03506425
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard S Bedlack, MD PhD
• Affiliation: Duke ALS Clinic

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Lab supported probable or more definite ALS by El Escorial Criteria

2. Age greater than or equal to 18 years

3. Willing and able to provide informed consent

4. On riluzole at a stable dose for at least 30d or not taking this

5. On Radicava at a stable dose for at least 30d or not taking this

6. Life expectancy at least 6 months

7. Currently managed on a reasonably stable diet, avoidance of fasting, carnitine or
medium chain triglyceride (MCT) oils

8. Must stop any other experimental ALS treatment for at least 30 days prior to screening

9. If sexually active, must agree to use contraceptive or abstinence for duration of
treatment with triheptanoin

10. Females of child bearing age must have negative pregnancy test at screening

Exclusion Criteria:

1. Unwilling or unable to provide informed consent

2. Previous intolerance or adverse reaction to triheptanoin or MCT

3. Conditions that will prohibit MRI scanning (metal in eye, some surgical implants,
claustrophobia, inability to lie supine)

4. 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

ALS

Intervention(s)

Drug: Triheptanoin

Primary Outcome(s)

ALS Functional Rating Scale-revised Version (ALSFRS-R) Slope [Time Frame: baseline, 6 months]

Secondary Outcome(s)

Change in NAA/Cr Ratio From Motor Cortex as Measured by Magnetic Resonance Spectroscopy [Time Frame: baseline, 6 months]

Change in Urine Isoprostane Levels, an Oxidative Stress Marker [Time Frame: baseline, 1 month]

Secondary ID(s)

Pro00092250

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ultragenyx Pharmaceutical Inc

Results

• Results available: Yes
• Date Posted: 24/12/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03506425