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Trial record 2 of 29 for:    amyotrophic lateral sclerosis duke

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

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ClinicalTrials.gov Identifier: NCT03506425
Recruitment Status : Active, not recruiting
First Posted : April 24, 2018
Last Update Posted : October 17, 2018
Sponsor:
Collaborator:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Richard Bedlack, M.D., Ph.D., Duke University

Brief 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.

Condition or disease Intervention/treatment Phase
ALS Drug: Triheptanoin Phase 1 Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
Actual Study Start Date : June 21, 2018
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : February 2019


Arm Intervention/treatment
Experimental: Group 1
Standard care for 1 month, then standard care and Triheptanoin for 5 months.
Drug: Triheptanoin
Triheptanoin is a medium chain triglyceride (MCT) that 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. The Triheptanoin we will use is a colorless to light yellow oil. The target triheptanoin dose for this study is 1g/kg/d. This target dose was selected because it was safe and tolerable and altered brain MR spectroscopy in patients with Huntington's Disease.

Experimental: Group 2
Standard care and Triheptanoin for 6 months.
Drug: Triheptanoin
Triheptanoin is a medium chain triglyceride (MCT) that 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. The Triheptanoin we will use is a colorless to light yellow oil. The target triheptanoin dose for this study is 1g/kg/d. This target dose was selected because it was safe and tolerable and altered brain MR spectroscopy in patients with Huntington's Disease.

No Intervention: Group 3
Healthy controls for biomarkers



Primary Outcome Measures :
  1. ALSFRS-R slope on treatment compared to pre-treatment [ Time Frame: 6 months ]
    The primary analysis is the slope of the revised ALS functional rating scale (ALSFRS-R) during treatment compared to pre-treatment. Pre-treatment slope for each participant will be estimated as follows: (48-enrollment ALSFRS-R)/months since symptom onset. This frequently used "pre-slope" method is simple and inexpensive, and can predict disease progression as well as more complicated and expensive tools, at least for periods of less than 1 year.


Secondary Outcome Measures :
  1. Magnetic Resonance Spectroscopy [ Time Frame: 6 months ]
    Effects of time, Triheptanoin on NAA/Cr ratio from motor cortex

  2. Oxidative Stress Markers [ Time Frame: 6 months ]
    Effects of time, ALS, Triheptanoin on urine Markers of Oxidative Stress



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03506425


Locations
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Richard Bedlack, M.D., Ph.D.
Ultragenyx Pharmaceutical Inc
Investigators
Principal Investigator: Richard S Bedlack, MD PhD Duke ALS Clinic

Responsible Party: Richard Bedlack, M.D., Ph.D., MD, PhD, Duke University
ClinicalTrials.gov Identifier: NCT03506425     History of Changes
Other Study ID Numbers: Pro00092250
First Posted: April 24, 2018    Key Record Dates
Last Update Posted: October 17, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: There is no plan to share data from this small pilot trial

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases