Safety and Tolerability of the Ketogenic Diet in ALS

Study Purpose:

This research is being done to see if the ketogenic diet (which is high in fat and low in carbohydrates) is safe and tolerable in amyotrophic lateral sclerosis (ALS) patients who are fed through a gastrostomy tube. This is not a study to see if ketogenic diets are effective in the treatment of ALS.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:

Dietary Supplement

Intervention Name:




Phase 3

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



Neals Affiliated?


Coordinating Center Contact Information

Johns Hopkins ALS Clinic

5801 Smith Avenue | McAuley Suite 110
Baltimore, Maryland, 21209 United States

Full Study Summary:

Ketogenic diets have been used for decades to treat intractable seizures. The mechanism conferring anticonvulsant activity is uncertain, but one possible mechanism may be related to hyperpolarization resulting from increased levels of ATP. Ketogenic diets have never been used in patients with ALS. Therefore the specific aims of our study are the following: 1. To determine if gastrostomy-fed ALS patients adhering to the 4:1 ketogenic diet (ratio of grams of fat to grams of protein plus carbohydrate) is safe and tolerable. We will determine safety and tolerance by measuring incidence and severity of adverse effects as well as careful monitoring of weight. 2. To evaluate changes in strength, fatigue and cognitive function while strictly adhering to the ketogenic diet. 3. To evaluate the changes in the percentage of body fat during the course of the study to determine the metabolic impact of the ketogenic diet. If safety and tolerability are established, further studies to determine efficacy of the ketogenic diet in patients with ALS will be planned.

Study Sponsor:

Johns Hopkins University

Estimated Enrollment:


Estimated Study Start Date:

10 / 31 / 2009

Estimated Study Completion Date:

01 / 01 / 2012

Posting Last Modified Date:

04 / 01 / 2015

Date Study Added to

11 / 19 / 2009

Minimum Age:

18 Years

Maximum Age:


Inclusion Criteria:

1. Familial or sporadic ALS diagnosed as probable, laboratory-supported probable or definite according to the World Federation of Neurology El Escorial criteria

2. Age 18 or older

3. Capable of providing informed consent and complying with trial procedures

4. Gastrostomy tube in place for the prior month

5. Appel ALS score less than 100

6. Able to stand on a scale with assistance

7. For patients with Appel ALS scores greater than 80, availability of caregiver who is willing and able to:

- Prepare, administer and log tube feeds

- Check and log gastric residuals

- Assist with weighing subject at home if necessary

8. Willing to chart food intake during the six-month study

9. Patients either not taking Riluzole (Rilutek) or Minocycline or on a stable dose of these for 30 days

10. Not taking Coenzyme Q10 or on a stable dose and brand for 30 days

11. Absence of exclusion criteria

Exclusion Criteria:

1. Forced vital capacity <50% of predicted

2. Dependence on mechanical ventilation for more than 12 hours per day

3. Exposure to any experimental agent within 30 days of onset of this protocol

4. Women who are pregnant or planning to become pregnant

5. Women of childbearing potential not practicing contraception

6. Enrollment in another research study within 30 days of or during this trial

7. Mini-Mental State Exam (MMSE) score <20

8. Patients with symptomatic cardiac disease or hypercholesterolemia

9. Patients with myocardial infarction within 6 months of this trial

10. Renal dysfunction defined as BUN and creatinine >2XULN

11. Known mitochondrial disease

12. BMI<18.5

13. Prior use of a 4:1 ketogenic diet or Atkins diet within 1 month of this trial

14. Impaired liver function, defined as AST or ALT of 3 X ULN

15. Patients who have a pacemaker or other internal electronic medical device

Johns Hopkins ALS Clinic

Baltimore, Maryland 21287
United States