CClinicalTrials.gg
CompletedNCT01716468Updated May 9, 2016Results posted

Ketogenic Diet in Advanced Cancer

An interventional study of Ketogenic Diet in Cancer, sponsored by VA Pittsburgh Healthcare System. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-05-09.

Sponsored by VA Pittsburgh Healthcare System · Not applicable and Interventional

Phase
Not applicable
Study type
Interventional
Enrollment
17
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

The purpose of this study is to determine the safety and tolerability of a modified low carbohydrate diet, determine if quality of life is improved and to determine if the diet has any effect on tumor growth (size or spread) and or to determine if there is any effect on progression free survival (PFS) and overall survival (OS) of subjects enrolled in the study.

Read the detailed description

In their natural state within the body, cells normally depend on cellular respiration as the source of energy and ATP. During times of complete or partial food deprivation these cells can also rely on ketosis as an additional energy source. Tumor cells in contrast are defective in this respect, lacking in the ability to utilize ketone bodies for energy and instead are heavily dependent on glycolysis for their survival. Malignant cells depend heavily on glycolysis for energy production even in high oxygen states. If the investigators limit the supply of glucose, glycolysis would decrease and tumor cells would lose their main source of energy supply.

In addition, increased carbohydrate intake is sensed by the pancreas and results in increased insulin secretion. Insulin is important for normal growth in the body and is a potent growth factor stimulating mitosis via signal transduction and DNA synthesis. It is a potent growth factor which acts by binding to cell membrane insulin receptors and stimulates mitosis via protein kinase B/Akt- mediated signal transduction and DNA synthesis.

Insulin also has anti-apoptotic properties, meaning less tumor cell death. Increased circulating insulin levels have been associated with increasing risk of certain cancers (i.e. prostate and colon cancer), but no association with serum glucose levels (8). By limiting carbohydrate intake, the investigators might expect decreased tumor growth and increased tumor cell destruction.

Most human and mouse tumors take up more glucose than their surrounding normal tissue. By using positron emission tomography with fluoro-deoxy-glucose, FDG-PET scans can be used to detect and monitor tumor response to chemotherapy and radiation.

In this study, PET/CT scanning would be utilized to measure the amount of (at the screening visit) glucose activity within certain tumors, before and after the diet.

Primary Aim: To determine the safety and tolerability of a modified low carbohydrate diet in people with advanced cancer across different tumor types.

Hypothesi(e)s: The long term tolerability of the KD (Ketogenic Diet) has been established (Groesbeck et al., 2006). Recent studies involving human patients with brain cancer showed tolerability of the Ketogenic diet over a period as long as 19 months with minimal side effects. However the MAD (Modified Atkins Diet) long term side effects are as of yet unclear. Given the similarity of the MAD compared to the KD, and being less restrictive, the long term side effects (kidney stones, dyslipidemia, gastroesophageal reflux) of the MAD is projected to be similar, if not reduced, in comparison to the KD. Modified carbohydrate diets as well as ketogenic diets have been tolerated by people with epilepsy. It is hypothesized that the effect this diet will have on overall weight loss, hyperlipidemia, and blood glucose levels will be minimal and tolerable even by cancer patients over a prolonged period of time, up to 12 months or possibly longer. Given the tolerability of the diet in brain cancer patients, it is hypothesized that patients with other types of tumors will find this diet tolerable and safe.

Secondary Aims: To determine if quality of life is improved with the use of this diet. To determine if the diet has any effect on tumor growth (size or spread) or to determine if there is any effect on progression free survival (PFS) and overall survival (OS) of subjects enrolled in the study.

Hypothesis(e): The diet showed good tolerance in human adult patient studies wherein the diet was tolerated well up to 12 weeks in a group of diverse tumor types and up to more than 10 years in a glioma patient. It is hypothesized that cancer patients will be able to tolerate this diet at 16 weeks or more. Studies in animals as well as in tumor tissue models show slowing of tumor growth, and in some instances also showed tumor shrinkage. Human case reports and a small study of 16 patients show tumor shrinkage and stable disease while on the diet.

02

Conditions studied

  • Cancer

Keywords

  • Ketogenic Diet, Advanced Cancer, Neoplasm
  • Low carbohydrate
03

In context

Lead sponsor

VA Pittsburgh Healthcare System is the lead sponsor of 19 studies on the registry; 3 are open to participants now.

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

Eligibility criteria

Subjects must meet all of the following criteria to be eligible for the study:

  • 18 years of age or older.
  • English speaking and written informed consent obtained.
  • Patients presenting to the VAPHS Oncology inpatient or outpatient clinic with cancer of advanced or metastatic stage.
  • Must have measurable disease at least 1 cm in greatest diameter on CT or MRI scanning. If a new FDG avid satellite lesions are noted this will be counted as disease progression. The minimum size is 1 cm. The tumor will be measured at the longest diameter. Either the primary tumor or metastatic tumor, either may be used. In absence of primary tumor, we usually measure the next largest metastatic lesion. The tumor will be chose by using RECIST criteria measure (at the screening visit) tumors, then using the 4 response criteria, CR complete response= disappearance of all target lesions, PR= 30% decrease in the sum of the longest diameter of target lesions, PD progressive disease= 20% increase in the sum of the longest diameter of target lesions, and SD stable disease= small changes that don't meet the above criteria
  • Patients must have solid, advanced or metastatic tumors, and have failed to respond to chemotherapy or on chemotherapy holiday.
  • Tumors must be PET positive in primary or metastatic site.
  • Patients must not be actively receiving chemotherapy after holiday or have refused or failed one or more prior chemotherapy or radiotherapy treatment.
  • Subjects may be enrolled in any other studies as long as they are not under an active form of chemotherapy or radiotherapy intervention.
  • No subjects will be excluded based on their race, religion, ethnicity, and gender or HIV status, as applicable.

Exclusion:

  • Prisoners or subjects who are involuntarily incarcerated.
  • Secondary brain metastases.
  • History of Gout.
  • AST or ATL > 1.5 X ULN.
  • Creatinine > 2.0.
  • Hematologic malignancies
  • BMI \< 22.
  • History of kidney disease or kidney stones.
  • Receiving chemotherapy including biologics.
  • Recent AMI/TIA or stroke within one month of study entry.
  • ECOG performance status of 3 or worse.
05

Study design

Phase
Not applicable
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
17 participants (actual)

Study arms

  • Other
    Advanced or metastatic cancer

    Patients chosen must be diagnosed with advanced or metastatic cancer of the following tumor types (colorectal, prostate, brain, breast, pancreatic, hepatobiliary, melanoma, sarcoma, non-small cell /small cell lung, genitourinary cancers).All participants will be assigned to a ketogenic diet. There are no randomization to other separate arms since this is a safety and feasibility study.

    Other: Ketogenic Diet

Interventions

  • OtherKetogenic Diet

    modified ketogenic diet

06

What researchers measure

Primary outcomes

  1. To Determine the Safety and Tolerability of a Modified Low Carbohydrate Diet in People With Advanced Cancer Across Different Tumor Types.

    Recent studies involving human patients with brain cancer showed tolerability of the Ketogenic diet over a period as long as 19 months with minimal side effects. It is hypothesized that the effect this diet will have on overall weight loss, hyperlipidemia, and blood glucose levels will be minimal and tolerable even by cancer patients over a prolonged period of time, up to 12 months or possibly longer. Serum fasting glucose, cholesterol, total, LDL, HDL and triglycerides, serum ketones in mg/dl units , weight in lbs. will be measured at designated time points. Number of patients actually tolerating the diet for at least 4 weeks or more will be recorded.

    Time frame: 16 weeks

07

Results

Posted May 9, 2016

Participant flow

Participant flow — Overall Study
MilestoneAdvanced or Metastatic Cancer
Started17
Completed11
Not completed6

Outcome measures

PrimaryTo Determine the Safety and Tolerability of a Modified Low Carbohydrate Diet in People With Advanced Cancer Across Different Tumor Types.

Recent studies involving human patients with brain cancer showed tolerability of the Ketogenic diet over a period as long as 19 months with minimal side effects. It is hypothesized that the effect this diet will have on overall weight loss, hyperlipidemia, and blood glucose levels will be minimal and tolerable even by cancer patients over a prolonged period of time, up to 12 months or possibly longer. Serum fasting glucose, cholesterol, total, LDL, HDL and triglycerides, serum ketones in mg/dl units , weight in lbs. will be measured at designated time points. Number of patients actually tolerating the diet for at least 4 weeks or more will be recorded.

Time frame:
16 weeks
Reported as:
Number · participants
To Determine the Safety and Tolerability of a Modified Low Carbohydrate Diet in People With Advanced Cancer Across Different Tumor Types.
participantsAdvanced or Metastatic Cancer
To Determine the Safety and Tolerability of a Modified Low Carbohydrate Diet in People With Advanced Cancer Across Different Tumor Types.11

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Advanced or Metastatic Cancer—0/11 (0%)7/11 (63.6%)
Most frequent other events
Most frequent other events
EventAdvanced or Metastatic Cancer
Grade 1 Weight LossGeneral disorders6/11
Grade 2 weight LossGeneral disorders1/11

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Advanced or Metastatic Cancer
<=18 years0
Between 18 and 65 years9
>=65 years8
Age, Continuous
Age, Continuous(years)Advanced or Metastatic Cancer
Mean65 (44 to 90)
Sex: Female, Male
Sex: Female, Male(Participants)Advanced or Metastatic Cancer
Female0
Male17
Region of Enrollment
Region of Enrollment(participants)Advanced or Metastatic Cancer
United States17
08

Study locations

1 site
  • VA Pittsburgh Healthcare System
    Pittsburgh, Pennsylvania 15240, United States
09

References and documents

Publications

  • Tan-Shalaby JL, Carrick J, Edinger K, Genovese D, Liman AD, Passero VA, Shah RB. Modified Atkins diet in advanced malignancies - final results of a safety and feasibility trial within the Veterans Affairs Pittsburgh Healthcare System. Nutr Metab (Lond). 2016 Aug 12;13:52. doi: 10.1186/s12986-016-0113-y. eCollection 2016. Erratum In: Nutr Metab (Lond). 2016 Sep 01;13(1):61. doi: 10.1186/s12986-016-0120-z. PubMed 27525031 ↗
10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 9, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01716468
Lead sponsor
VA Pittsburgh Healthcare System
Responsible party
Jocelyn Tan (Attending Physician-Hematology/Oncology, VA Pittsburgh Healthcare System) — Principal investigator
First posted
Oct 29, 2012
Start date
Oct 2012
Primary completion
Jan 2015
Completion
Dec 2015
Results posted
May 9, 2016
Last update
May 9, 2016

Study contacts

Jocelyn Tan, MD
principal investigator · VA Pittsburgh Healthcare System

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Apr 2016. You cannot join it, but the record below documents what was studied.

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