Safety and Tolerability of the Ketogenic Diet in Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 13 April 2015
• Main ID: NCT01016522
• Date of registration: 18/11/2009
• Prospective Registration: No
• Primary sponsor: Johns Hopkins University
• Public title: Safety and Tolerability of the Ketogenic Diet in Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: Safety and Tolerability of the Ketogenic Diet in ALS
• Date of first enrolment: November 2009
• Target sample size: 1
• Recruitment status: Terminated
• URL: http://clinicaltrials.gov/show/NCT01016522
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

United States

Key inclusion & exclusion criteria

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



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

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Dietary Supplement: KetoCal

Primary Outcome(s)

Study will determine if humans with ALS fed a strictly controlled diet designed to generate large amounts of ketones is safe and well-tolerated without evidence of weight loss or other adverse effect [Time Frame: 28 weeks]

Secondary Outcome(s)

Evaluate changes in motor function, strength, fatigue, body fat and cognitive function [Time Frame: 28 weeks]

Secondary ID(s)

NA_00008855

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Nutricia North America

Cornell University