About this trial
The aim of this study is to evaluate the potential of BHB supplementation as a novel strategy to impede the development and progression of intestinal adenomas in individuals with FAP, thus potentially reducing the need for frequent upper endoscopies and colonoscopies and preventing the need for risk-reducing surgical intervention.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Have a diagnosis of FAP with genetic testing demonstrating a pathogenic or likely pathogenic germline variant in APC, must have a clinical FAP phenotype with at least one member of the family who has a pathogenic or likely pathogenic germline variant in APC, or must have a clinical diagnosis of FAP as agreed by two gastrointestinal cancer genetics experts
Must have an extensive colonic resection with either a subtotal colectomy with ileorectal anastomosis (STC-IRA) or total proctocolectomy with ileal pouch anal anastomosis (TPC-IPAA)
Can provide informed consent
Disqualifiers
Subject is pregnant, a prisoner, or is under 18 years of age
Prior total proctocolectomy with end ileostomy
History of inflammatory bowel disease
History of diabetes mellitus and are currently on medical diabetes therapy
Trial design
Single group
Treatments tested in this trial
R-1,3-Butanediol (10G-A)
Dietary supplementStudy participants will take one 35mL dose of HVMN Ketone-IQ by mouth per day (10 total grams of R-1,3-Butanediol) for 2 weeks
R-1,3-Butanediol (20G-A)
Dietary supplementStudy participants will take two 35mL dose of HVMN Ketone-IQ by mouth per day (20 total grams of R-1,3-Butanediol) for 2 weeks
R-1,3-Butanediol (30G-A)
Dietary supplementStudy participants will take three 35mL dose of HVMN Ketone-IQ by mouth per day (30 total grams of R-1,3-Butanediol) for 2 weeks
R-1,3-Butanediol (30G-B)
Dietary supplementStudy participants will take one 35mL dose of HVMN Ketone-IQ by mouth three times per day (30 total grams of R-1,3-Butanediol) for 12 weeks, with a possible additional 12 week extension
Treatment groups
Trial outcomes
Primary outcomes
Determine whether oral BHB supplementation is safe and tolerable in FAP
Assessment of FAP patient tolerance of BHB supplements through monitoring of side effects and/or intolerances and patient compliance. We will monitor the percentage of individuals who continue BHB supplementation for the duration of the study, as well as the compliance with taking the BHB supplement.
Secondary outcomes
Measure whether oral BHB supplementations increases serum BHB levels in FAP
Measure fasting serum BHB levels before, during, and at the end of BHB supplementation to determine if BHB supplementation significantly increases serum BHB levels.
Change in transcription and protein expression in the intestinal mucosa and in intestinal polyps in FAP after oral BHB supplementation
Transcriptional profiling and subsequent validation with protein level determination will be performed in the intestinal mucosa and in intestinal polyps before and after BHB supplementation to determine if BHB supplementation is having measurable transcriptional and protein effects on the intestinal mucosa and on intestinal polyps.
Determine whether oral BHB supplementation in FAP reduces intestinal polyp burden
The polyp burden in the duodenum as well as in the lower GI tract (rectal cuff, rectum, rectal pouch) will be calculated through polyp counting and measurement on endoscopy before and after BHB supplementation to permit comparison of polyp burden before and after BHB supplementation.
Sponsors and contacts
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Abramson Cancer Center at Penn Medicine
Lead sponsor
The V Foundation for Cancer Research
Collaborator