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

Safety and Tolerability of the Ketogenic Diet in ALS

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 Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Dietary supplement: KetoCal

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins ALS Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: KetoCal; KetoCal tube feeding formula	Dietary supplement: KetoCal; Ketogenic Diet food via gastrostomy tube - 80% fat, 17% protein, 3% carbohydrates

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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	28 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Evaluate changes in motor function, strength, fatigue, body fat and cognitive function	28 weeks

Collaborators and Investigators

• Sponsor: Johns Hopkins University
• Collaborators: Cornell University; Nutricia North America

Study record dates

Study Major Dates

• Study Start: November 1, 2009
• Primary Completion (Actual): February 1, 2011
• Study Completion (Actual): January 1, 2012

Study Registration Dates

• First Submitted: November 18, 2009
• First Submitted That Met QC Criteria: November 18, 2009
• First Posted (Estimate): November 19, 2009

Study Record Updates

• Last Update Posted (Estimate): April 1, 2015
• Last Update Submitted That Met QC Criteria: March 30, 2015
• Last Verified: March 1, 2015

More Information

Terms related to this study

• Keywords: ALS; Ketogenic Diet
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: NA_00008855