- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02716662
Safety of Caprylic Triglycerides in ALS: A Pilot Study
April 25, 2018 updated by: Dr. Dale J. Lange, Weill Medical College of Cornell University
The purpose of this study is to determine if AC-1204 is safe and tolerated in subjects with ALS.
The reason why the investigator wants to use AC-1204 in patients with ALS is to determine if, by taking AC-1204, the body will make substances called ketone bodies.
Further, if AC-1204 is well tolerated, the investigators want to change the amount the participant takes, to determine if the amount of ketone bodies in the blood increase in accordance with increases in the amount of AC-1204 the participant takes.
The investigators want to do this study because when the investigators gave AC-1204 to mice with ALS, findings suggest the disease course is altered for the better and that the cause of this change is due to the presence of ketones in the blood.
If AC-1204 can be proven to be safe and able to cause ketones to increase in the blood, the investigators will likely do subsequent studies to determine if the presence of ketone bodies will slow or stop the progression of the disease.
However, this study is not designed to determine if AC-1204 will stop or slow the progression of ALS.
It is designed to only determine if patients with ALS can tolerate AC-1204, if ketone bodies are produced and if the amount of ketone bodies produced increases with increasing dose.
Study Overview
Study Type
Interventional
Enrollment (Anticipated)
10
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
New York
-
New York, New York, United States, 10021
- Weill Cornell Medical Center
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 99 years (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Familial or sporadic ALS diagnosed as probable, laboratory-supported probable or definite according to the World Federation of Neurology El Escorial criteria,
- Age 18 or older,
- Capable of providing informed consent and complying with trial procedures,
- Appel ALS score less than 100,
- Able to stand on a scale with assistance,
- Willing to chart food intake during the 12 week study,
- Patients either not taking Riluzole (Rilutek) or on a stable dose for 30 days or more,
- Not taking Coenzyme Q10 or on a stable dose and brand for 30 days,
- Absence of exclusion criteria.
Exclusion Criteria:
- Forced vital capacity <40% of predicted,
- Dependence on mechanical ventilation for more than 12 hours per day,
- Exposure to any experimental agent within 30 days of entry or at any time during the trial,
- Women who are breastfeeding, who are pregnant or are planning to become pregnant,
- Women of childbearing potential not practicing a medically accepted form of contraception,
- Enrollment in another research study within 30 days of or during this trial,
- Mini-Mental State Exam (MMSE) score <20,
- Patients with symptomatic cardiac disease or hypercholesterolemia,
- Patients with myocardial infarction within 6 months of this trial,
- Renal dysfunction defined as BUN and creatinine >2xULN,
- Known mitochondrial disease,
- BMI<18.5,
- Prior use of a 4:1 ketogenic diet or Atkins diet within 1 month of this trial,
- Impaired liver function, defined as AST or ALT of 3xULN,
- Patients who have a pacemaker or other internal electronic medical device,
- Known allergy or hypersensitivity to milk or soy products.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Open Label
Axona
|
medical powder, supplement food
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Ketone levels
Time Frame: 4 months
|
4 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
January 1, 2016
Primary Completion (ACTUAL)
May 1, 2017
Study Completion (ACTUAL)
April 25, 2018
Study Registration Dates
First Submitted
January 6, 2016
First Submitted That Met QC Criteria
March 17, 2016
First Posted (ESTIMATE)
March 23, 2016
Study Record Updates
Last Update Posted (ACTUAL)
April 27, 2018
Last Update Submitted That Met QC Criteria
April 25, 2018
Last Verified
April 1, 2018
More Information
Terms related to this study
Other Study ID Numbers
- 1307014081
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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