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CompletedNCT00983983Updated Feb 27, 2015Results posted

High Fat/High Calorie Trial in Amyotrophic Lateral Sclerosis

A Phase 2 interventional study of Oxepa and Jevity 1.5 in Amyotrophic Lateral Sclerosis, sponsored by Massachusetts General Hospital. Completed at 13 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2015-02-27.

Sponsored by Massachusetts General Hospital · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
28
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

The purpose of this study is to determine the safety, tolerability, and preliminary efficacy of long-term use of high fat/high calorie and high calorie diets in people with amyotrophic lateral sclerosis (ALS) (Lou Gehrig's disease).

Read the detailed description

Weight loss is a common and severe symptom of amyotrophic lateral sclerosis (ALS), caused both from inadequate calorie intake and an increased metabolic rate. People with ALS are generally instructed to increase their calorie intake; however, the ideal amount and type of calories has not been studied. Several studies in an animal model of motor neuron disease have shown that a high fat/high calorie diet can increase survival by as much as 38%. Mice on a high fat diet also live longer than mice fed diets consisting of high protein or high sugar. We are therefore conducting a phase II safety, tolerability, and preliminary efficacy trial in ALS of high fat versus high calorie versus normal diet. The normal diet will be calculated based on the number of calories needed to replace each participant's measured daily calorie requirement.

02

Conditions studied

  • Amyotrophic Lateral Sclerosis

Keywords

  • Amyotrophic Lateral Sclerosis
  • ALS
  • Motor Neuron Disease
  • MND
  • Fat
  • Lipid
  • Cholesterol
  • Omega-3 fatty acid
  • Diet
  • Tube feed
  • Gastrostomy
  • PEG
  • Adults with Amyotrophic Lateral Sclerosis (ALS)
03

In context

Amyotrophic Lateral Sclerosis

1,000 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 284 are open to participants now.

This study's enrollment of 28 is below the median of 37 across 678 interventional studies indexed under Amyotrophic Lateral Sclerosis.

Browse Amyotrophic Lateral Sclerosis studies →

Lead sponsor

Massachusetts General Hospital is the lead sponsor of 2,582 studies on the registry; 451 are open to participants now.

Of its 221 completed or terminated interventional studies of FDA-regulated products, 168 (76%) have results posted.

Browse Massachusetts General Hospital studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Clinical diagnosis of ALS
  2. Male or female subjects aged 18 years or older
  3. Must already be tolerating tube feedings through either a gastrostomy tube (G-tube or PEG) or jejunostomy tube (J-tube)
  4. Must require non-invasive ventilation (BIPAP) for less than 10 hours/day
  5. Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating.

Exclusion criteria

Exclusion Criteria:

  1. History of hepatitis including non-alcoholic steatohepatitis (NASH), cholecystectomy, prior biliary disease such as gallstones
  2. History of diabetes
  3. History of prior myocardial infarction or stroke
  4. Laboratory values: Screening alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2.0 times the upper limit of normal or total bilirubin greater than 1.5 times the upper limit of normal
  5. Allergy to soy, fish, or milk products
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
28 participants (actual)

Study arms

  • Experimental
    High fat/high calorie

    High fat/high calorie diet: Oxepa

    Dietary Supplement: Oxepa

  • Active comparator
    High calorie

    High calorie diet: Jevity 1.5

    Dietary Supplement: Jevity 1.5

  • Placebo comparator
    Control

    Control diet: Jevity 1.0

    Dietary Supplement: Jevity 1.0

Interventions

  • Dietary supplementOxepa

    Oxepa: Tube feed containing 1.5 calories/ml of which 55% calories are from fat, including eicosapentaenoic acid and gamma-linolenic acid. Subjects will receive 1.25 times their daily caloric requirements based on their measured resting energy expenditure. Subjects will receive 4 months of tube feeds and be followed for an additional 1 month to measure adverse events and tolerability.

  • Dietary supplementJevity 1.5

    Jevity 1.5: Tube feed containing 1.5 calories/ml of which 29.4% are from fat. Subjects will receive 1.25 times their daily caloric requirements based on their measured resting energy expenditure. Subjects will receive 4 months of tube feeds and be followed for an additional 1 month to measure adverse events and tolerability.

  • Dietary supplementJevity 1.0

    Jevity 1.0: Control tube feed. Subjects will receive 1.0 times their daily caloric requirements based on their measured resting energy expenditure. Subjects will receive 4 months of tube feeds and be followed for an additional 1 month to measure adverse events and tolerability.

06

What researchers measure

Primary outcomes

  1. Safety Outcomes: Frequency of Adverse Events

    Time frame: 5 months

  2. Serious Adverse Events

    SAE were defined using the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0.

    Time frame: 5 months

  3. Tolerability

    Number of participants who completed the study on their assigned study intervention.

    Time frame: 5 months

Secondary outcomes

  1. Rate of Change in ALSFRS-R in Units/Month

    Rate of change in the ALS Functional Rating Scale-Revised, calculated in units/month. Negative numbers refer to worsening over time.

    Time frame: Over 5 months

  2. Biomarkers of Body Composition and Lipid Metabolism

    Time frame: 5 months follow-up

07

Results

Posted Feb 27, 2015

Participant flow

Participant flow — Overall Study
MilestoneHigh Fat/High CalorieHigh CalorieControl
Started897
Completed686
Not completed211

Outcome measures

SecondaryRate of Change in ALSFRS-R in Units/Month

Rate of change in the ALS Functional Rating Scale-Revised, calculated in units/month. Negative numbers refer to worsening over time.

Time frame:
Over 5 months
Reported as:
Mean · units on a scale/month
Rate of Change in ALSFRS-R in Units/Month
units on a scale/monthHigh Fat/High CalorieHigh Calorie/High CarbohydrateControl
Rate of Change in ALSFRS-R in Units/Month-1.54 (-2.36 to -0.73)-1.07 (-1.71 to -0.42)-2.17 (-3.24 to -1.10)
Statistical analysis
  • High Calorie/High Carbohydrate vs Control · Mixed Models Analysis · p = 0.07
SecondaryBiomarkers of Body Composition and Lipid Metabolism
Time frame:
5 months follow-up

Results for this outcome have not been posted.

PrimarySafety Outcomes: Frequency of Adverse Events
Time frame:
5 months
Reported as:
Number · Total Number of Adverse Events
Safety Outcomes: Frequency of Adverse Events
Total Number of Adverse EventsHigh Fat/High CalorieHigh CalorieControl
Safety Outcomes: Frequency of Adverse Events492442
Statistical analysis
  • High Calorie vs Control · Fisher Exact · p = 0.06
PrimarySerious Adverse Events

SAE were defined using the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0.

Time frame:
5 months
Reported as:
Number · Number of Serious Adverse Events
Serious Adverse Events
Number of Serious Adverse EventsHigh Fat/High CalorieHigh CalorieControl
Serious Adverse Events309
Statistical analysis
  • High Calorie vs Control · Fisher Exact · p = 0.0005
PrimaryTolerability

Number of participants who completed the study on their assigned study intervention.

Time frame:
5 months
Reported as:
Number · participants
Tolerability
participantsHigh Fat/High CalorieHigh CalorieControl
Tolerability571
Statistical analysis
  • High Calorie vs Control · Fisher Exact · p = 0.03

Adverse events

Collected over Participants were followed for adverse events for 30 days after stopping study diet.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
High Fat/High Calorie—3/6 (50%)6/6 (100%)
High Calorie/High Carbohydrate—0/8 (0%)8/8 (100%)
Control—4/6 (66.7%)6/6 (100%)
Most frequent serious events
Most frequent serious events
EventHigh Fat/High CalorieHigh Calorie/High CarbohydrateControl
AspirationRespiratory, thoracic and mediastinal disorders0/60/83/6
Dyspnea (shortness of breath)Respiratory, thoracic and mediastinal disorders2/60/81/6
HypoxiaRespiratory, thoracic and mediastinal disorders0/60/81/6
Stomach PainGastrointestinal disorders0/60/81/6
Infection-LungInfections and infestations0/60/81/6
DepressionPsychiatric disorders1/60/80/6
Most frequent other events
Showing 10 of 50
Most frequent other events
EventHigh Fat/High CalorieHigh Calorie/High CarbohydrateControl
DiarrheaGastrointestinal disorders4/61/83/6
ConstipationGastrointestinal disorders3/60/80/6
Bloating/FlatulenceGastrointestinal disorders1/61/83/6
FatigueGeneral disorders1/61/82/6
Infection-Urinary TractInfections and infestations1/61/82/6
Dyspnea-Shortness of BreathRespiratory, thoracic and mediastinal disorders2/61/81/6
Infection-Lung (Pneumonia)Infections and infestations1/60/82/6
Oral/Perioral InfectionInfections and infestations2/61/80/6
Pain-Abdomen/StomachGastrointestinal disorders2/61/81/6
NauseaGastrointestinal disorders1/62/81/6

Baseline characteristics

Age, Continuous
Age, Continuous(years)High Fat/High CalorieHigh Calorie/High CarbohydrateControlTotal
Mean64.0 ± 6.957.5 ± 15.463.2 ± 9.461.3 ± 11.4
Sex: Female, Male
Sex: Female, Male(Participants)High Fat/High CalorieHigh Calorie/High CarbohydrateControlTotal
Female55111
Male34613
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)(units on a scale)High Fat/High CalorieHigh Calorie/High CarbohydrateControlTotal
Mean30.7 ± 7.025.5 ± 9.423.0 ± 3.526.3 ± 7.6
08

Study locations

13 sites
  • Barrow Neurological Institute/St. Joseph's Hospital and Medical Center
    Phoenix, Arizona 85013, United States
  • University of California at Irvine
    Irvine, California 92868, United States
  • California Pacific Medical Center, University of California at San Francisco
    San Francisco, California 94120, United States
  • Sarasota Memorial Hospital
    Sarasota, Florida 34239, United States
  • Emory University School of Medicine
    Atlanta, Georgia 30322, United States
  • Neurology Clinical Trials Unit, Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • Saint Mary's Health Care
    Grand Rapids, Michigan 49503, United States
  • Columbia Presbyterian Medical Center
    New York, New York 10032, United States
  • Carolinas Medical Center Neuromuscular/ALS-MDA Center
    Charlotte, North Carolina 28207, United States
  • Oregan Health and Science University
    Portland, Oregon 97239, United States
  • Drexel University
    Philadelphia, Pennsylvania 19107, United States
  • Methodist Neurological Institute
    Houston, Texas 77030, United States
  • University of Vermont
    Burlington, Vermont 05401, United States
09

References and documents

Publications

  • Kasarskis EJ, Berryman S, Vanderleest JG, Schneider AR, McClain CJ. Nutritional status of patients with amyotrophic lateral sclerosis: relation to the proximity of death. Am J Clin Nutr. 1996 Jan;63(1):130-7. doi: 10.1093/ajcn/63.1.130. PubMed 8604660 ↗
  • Desport JC, Torny F, Lacoste M, Preux PM, Couratier P. Hypermetabolism in ALS: correlations with clinical and paraclinical parameters. Neurodegener Dis. 2005;2(3-4):202-7. doi: 10.1159/000089626. PubMed 16909026 ↗
  • Desport JC, Preux PM, Magy L, Boirie Y, Vallat JM, Beaufrere B, Couratier P. Factors correlated with hypermetabolism in patients with amyotrophic lateral sclerosis. Am J Clin Nutr. 2001 Sep;74(3):328-34. doi: 10.1093/ajcn/74.3.328. PubMed 11522556 ↗
  • Morozova N, Weisskopf MG, McCullough ML, Munger KL, Calle EE, Thun MJ, Ascherio A. Diet and amyotrophic lateral sclerosis. Epidemiology. 2008 Mar;19(2):324-37. doi: 10.1097/EDE.0b013e3181632c5d. PubMed 18300717 ↗
  • Veldink JH, Kalmijn S, Groeneveld GJ, Wunderink W, Koster A, de Vries JH, van der Luyt J, Wokke JH, Van den Berg LH. Intake of polyunsaturated fatty acids and vitamin E reduces the risk of developing amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2007 Apr;78(4):367-71. doi: 10.1136/jnnp.2005.083378. Epub 2006 Apr 28. PubMed 16648143 ↗
  • Mattson MP, Cutler RG, Camandola S. Energy intake and amyotrophic lateral sclerosis. Neuromolecular Med. 2007;9(1):17-20. doi: 10.1385/nmm:9:1:17. PubMed 17114821 ↗
  • Dupuis L, Oudart H, Rene F, Gonzalez de Aguilar JL, Loeffler JP. Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high-energy diet in a transgenic mouse model. Proc Natl Acad Sci U S A. 2004 Jul 27;101(30):11159-64. doi: 10.1073/pnas.0402026101. Epub 2004 Jul 19. PubMed 15263088 ↗
  • Wills AM, Hubbard J, Macklin EA, Glass J, Tandan R, Simpson EP, Brooks B, Gelinas D, Mitsumoto H, Mozaffar T, Hanes GP, Ladha SS, Heiman-Patterson T, Katz J, Lou JS, Mahoney K, Grasso D, Lawson R, Yu H, Cudkowicz M; MDA Clinical Research Network. Hypercaloric enteral nutrition in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet. 2014 Jun 14;383(9934):2065-2072. doi: 10.1016/S0140-6736(14)60222-1. Epub 2014 Feb 28. PubMed 24582471 ↗
  • Paganoni S, Deng J, Jaffa M, Cudkowicz ME, Wills AM. Body mass index, not dyslipidemia, is an independent predictor of survival in amyotrophic lateral sclerosis. Muscle Nerve. 2011 Jul;44(1):20-4. doi: 10.1002/mus.22114. Epub 2011 May 23. PubMed 21607987 ↗
10

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Oct 2009, a primary completion of Apr 2013 and a completion of May 2013. It does not say whether these dates are actual or estimated.

Results were first posted in Feb 2015, 1 year 10 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 23, 2009
First posted
Sep 24, 2009
Start
Oct 2009
Primary completionActual
Apr 2013
CompletionActual
May 2013
Results first posted
Feb 27, 2015
Last update
Feb 27, 2015
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Feb 27, 2015, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00983983
Lead sponsor
Massachusetts General Hospital
Collaborators
Muscular Dystrophy Association
Responsible party
Anne-Marie Alexandra Wills, MD (Assistant Professor of Neurology, Massachusetts General Hospital) — Principal investigator
First posted
Sep 24, 2009
Start date
Oct 2009
Primary completion
Apr 2013
Completion
May 2013
Results posted
Feb 27, 2015
Last update
Feb 27, 2015

Study contacts

Anne-Marie A Wills, M.D.
principal investigator · Massachusetts General Hospital

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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