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

• ClinicalTrials.gov Identifier: NCT01016522
• Recruitment Status: Terminated
• First Posted: November 19, 2009
• Last Update Posted: April 1, 2015
• Sponsor: Johns Hopkins University
• Collaborators: Nutricia North America; Cornell University
• Information provided by (Responsible Party): Vinay Chaudhry, Johns Hopkins University

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Amyotrophic Lateral Sclerosis	Dietary Supplement: KetoCal	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 1 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Safety and Tolerability of the Ketogenic Diet in ALS
• Study Start Date: November 2009
• Actual Primary Completion Date: February 2011
• Actual Study Completion Date: January 2012

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. 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 Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

United States, Maryland

Johns Hopkins ALS Clinic

Baltimore, Maryland, United States, 21287

Sponsors and Collaborators

Johns Hopkins University

Nutricia North America

Cornell University

More Information

• Responsible Party: Vinay Chaudhry, Professor, Johns Hopkins University
• ClinicalTrials.gov Identifier: NCT01016522
• Obsolete Identifiers: NCT01035710
• Other Study ID Numbers: NA_00008855
• First Posted: November 19, 2009
• Last Update Posted: April 1, 2015
• Last Verified: March 2015

Keywords provided by Vinay Chaudhry, Johns Hopkins University:

ALS; Ketogenic Diet

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases