β-Hydroxybutyrate suppresses colorectal cancer.
Oxana Dmitrieva-Posocco, Andrea C Wong, Patrick Lundgren...
https://pubmed.ncbi.nlm.nih.gov/35477756Actively Recruiting
Phase Not Applicable
Age: 18Years +
All Genders
ID06578637
Investigation of β-hydroxybutyrate Supplementation as Chemoprevention in Familial Adenomatous Polyposis
Led by Abramson Cancer Center at Penn Medicine · Updated on 2025-09-04
20
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
A
Abramson Cancer Center at Penn Medicine
Lead Sponsor
T
The V Foundation for Cancer Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the potential of beta-hydroxybutyrate (BHB) supplementation as a new way to slow the growth and development of intestinal adenomas in people with Familial Adenomatous Polyposis (FAP). This condition leads to numerous polyps in the intestines, increasing the risk of cancer. The study aims to reduce the need for frequent invasive procedures like upper endoscopies and colonoscopies, as well as prevent surgeries that lower cancer risk. The study includes two parts: Part A involves up to 9 participants with FAP taking different doses of an oral BHB precursor called R-1,3-butanediol (HVMN Ketone-IQ) over two weeks. Part B is a longer open-label study involving up to 20 participants who will undergo colonoscopy or sigmoidoscopy and upper endoscopy before starting daily BHB supplementation for 12 weeks, with a possible 12-week extension. Doses vary from one to three 35mL oral doses daily, delivering 10 to 30 grams of R-1,3-butanediol. Participants will have blood and stool samples collected at multiple points, including before and during treatment, to measure ketone levels and biological changes. Colonoscopies and upper endoscopies are performed at the start, after 12 weeks, and after 24 weeks if participants continue in the extension. The study will assess safety, tolerability, changes in ketone levels, intestinal mucosa and polyp characteristics, and changes in polyp burden over approximately three years.
CONDITIONS
Brief Title
Investigation of β-hydroxybutyrate Supplementation as Chemoprevention in Familial Adenomatous Polyposis
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Diagnosis of Familial Adenomatous Polyposis confirmed by genetic testing or clinical evaluation by experts
- Extensive colonic surgery with subtotal colectomy with ileorectal anastomosis or total proctocolectomy with ileal pouch anal anastomosis (Part A)
- Willingness to undergo colonoscopy/sigmoidoscopy and upper endoscopy with at least two colorectal polyps present at enrollment (Part B)
- Ability to provide informed consent
You will not qualify if you...
- Pregnancy, prisoner status, or under 18 years of age
- Prior total proctocolectomy with end ileostomy
- History of inflammatory bowel disease
- History of diabetes currently treated with medication
- Chronic kidney disease with eGFR less than 60 mL/min/1.73m2
- Active cancer treatment
- Use of ketogenic diet or intermittent fasting (16+ hours daily) in the 4 weeks before enrollment
- Regular use of certain chemopreventive agents including aspirin over 81mg daily, NSAIDs, BHB supplements, or other related medications within 6 weeks before enrollment (Part B)
- Presence of colonic or small intestinal polyps larger than 1 cm not removed (excluding ampullary adenomas) (Part B)
AI-Screening
AI-Powered Screening
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Screening and enrollment visit
Initial Absorption Study
Duration - 2 weeks
Participants take R-1,3-butanediol at one of three doses for 2 weeks with blood samples collected before and after dosing to study absorption.
2 visits (one before and one after dosing, in-person)
Longitudinal Open-Label Study Treatment
Duration - 12 weeks
Participants receive daily oral doses of R-1,3-butanediol for 12 weeks with periodic blood and stool sample collections and weekly home ketone monitoring.
3 visits (baseline, 4 weeks, and 8 weeks) plus weekly home ketone checks
Endoscopic Assessments
Duration - At baseline and after 12 weeks
Participants undergo colonoscopy/sigmoidoscopy and upper endoscopy at study entry and after 12 weeks of treatment to evaluate intestinal polyps.
2 visits (in-person endoscopic procedures)
Optional Extension Treatment
Duration - Up to 12 weeks
Participants may continue R-1,3-butanediol for an additional 12 weeks followed by repeat endoscopic assessments.
1 visit for endoscopic assessment after extension
Trial Site Locations
Total: 1 location
1
Abramson Cancer Center of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
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Research Team
B
Bryson W Katona, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
PREVENTION
Number of Arms
4
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