Efficacy of Vitamin B1 in Familial Adenomatous Polyposis Patients. (VitB1; FAP)

Evaluation of the Efficacy of Vitamin B1 in Patients With Familial Adenomatous Polyposis: An Exploratory Study

This study aimed to investigate the efficacy of vitamin B1 in patients with familial adenomatous polyposis, and to determine the inhibitory effect of vitamin B1 on the size and number of colorectal adenomas by administering oral vitamin B1 therapy for 1 year and colonoscopy follow-up every 3 months

Study Overview

• Status: Enrolling by invitation
• Conditions: Familial Adenomatous Polyposis (FAP)
• Intervention / Treatment: Drug: Vitamin B1

Detailed Description

The investigators will mark a region with a diameter of no more than 5 cm, containing at least 3 polyps, each with a diameter of 5-15 mm. Continuous video recording will be conducted throughout the colonoscopy procedure to provide baseline reference images of the colorectal polyps. Patients with familial adenomatous polyposis were given 100mg bid dose of vitamin B1 orally for one year, and colonoscopies were followed every 3 months to observe changes in the size and number of polyps in the designated area.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200032
      • Shanghai Zhongshan Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria

1. Patients with a confirmed diagnosis of classical familial adenomatous polyposis (FAP) based on colonoscopy, histopathology, and genetic testing.
2. Aged between 18 and 80 years, with no restriction on gender.
3. No history of total colectomy, with remaining bowel segments available for evaluation.
4. Presence of more than 10 adenomatous polyps in the colon or rectum.
5. Normal liver and kidney function.
6. Willing to participate in vitamin B1 therapy and commit to regular follow-up appointments.

Exclusion Criteria

1. Use of medications for more than one week in the past three months, or inability to avoid continuous use during the study period, that may affect colorectal polyps, including aspirin, nonsteroidal anti-inflammatory drugs (NSAIDs), ω-3 polyunsaturated fatty acids, rapamycin, and curcumin.
2. Use of medications for more than one week in the past three months, or inability to avoid continuous use during the study period, that may affect vitamin B1 absorption, metabolism, or excretion, including aminoglycoside antibiotics, thiazide diuretics, oral contraceptives, antiepileptic drugs, and antituberculosis drugs.
3. Attenuated familial adenomatous polyposis (AFAP).
4. Allergy to vitamin B1.
5. Contraindications to vitamin B1, such as liver or kidney dysfunction, or severe neuropsychiatric disorders.
6. History of total colectomy, with no remaining bowel segments.
7. Pregnant or breastfeeding individuals.
8. Presence of advanced colorectal cancer or metastatic cancer in other locations.
9. Previous radiotherapy, chemotherapy, or tumor immunotherapy.
10. History of alcohol or other substance abuse.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Vitamin B1	Drug: Vitamin B1; Vitamin B1 tablets, 100mg bid, taken orally for one year

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sum of polyp diameters in the delineated area	The diameter of each polyp was assessed by two independent endoscopists and the average of two diameters perpendicular to each other was reported. The diameter of the polyp was determined with reference to the titanium clip, blood vessels and scar in the visual field.	3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of polyps in the delineated area	The number of polyps was assessed by two independent endoscopists, and disputes were decided by a third endoscopist	3 months
Levels of vitamin B1-associated metabolites, including TMP, TPP, TTP, and free thiamine, in blood and urine samples and in polyps outside the delineated area	The content of vitamin B1 related metabolites was determined by liquid chromatography-tandem mass spectrometry	3 months

Collaborators and Investigators

• Sponsor: Shanghai Zhongshan Hospital
• Investigators: Principal Investigator: Pinghong Zhou, MD, PhD, Fudan University

Study record dates

Study Major Dates

• Study Start (Actual): January 8, 2024
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: October 18, 2024
• First Submitted That Met QC Criteria: October 18, 2024
• First Posted (Actual): October 21, 2024

Study Record Updates

• Last Update Posted (Actual): April 24, 2026
• Last Update Submitted That Met QC Criteria: April 21, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: thiamin; Familial adenomatous polyposis
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Genetic Diseases, Inborn; Intestinal Diseases; Neoplasms by Histologic Type; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Colorectal Neoplasms; Intestinal Neoplasms; Neoplasms, Glandular and Epithelial; Colonic Diseases; Adenoma; Neoplastic Syndromes, Hereditary; Adenomatous Polyps; Intestinal Polyposis; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Adenomatous Polyposis Coli; Sulfur Compounds; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Thiazoles; Azoles; Pyrimidines; Thiamine
• Other Study ID Numbers: ShanghaiZhongshanEndoscopy2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Original data could be shared at request by email after publication
• IPD Sharing Time Frame: The data will be available after publication of the study
• IPD Sharing Access Criteria: The shared data could be available by contacting Dr. Xin-Yang Liu at liu.xinyang@zs-hospital.sh.cn.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ANALYTIC_CODE; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No