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Familial Adenomatous Polyposis clinical trials

View clinical trials related to Familial Adenomatous Polyposis.

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NCT ID: NCT06308445 Not yet recruiting - Clinical trials for Familial Adenomatous Polyposis

Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis

RAPA-4-PAF
Start date: August 1, 2024
Phase: Phase 2
Study type: Interventional

The hypothesize of this research is that rapamycin is effective and well-tolerated in teenagers with familial adenomatous polyposis (FAP). Rapamycin could be effective in blocking the formation of adenomas and/or their evolution by decreasing their size and number. Researchers aim to assess the safety profile of rapamycin in FAP adolescents using a 2 low dose regimen.

NCT ID: NCT05630794 Not yet recruiting - Clinical trials for Colorectal Carcinoma

Testing ONC201 to Prevent Colorectal Cancer

Start date: October 8, 2024
Phase: Phase 1
Study type: Interventional

This phase I trial tests the safety, side effects, and best dose of Akt/ERK Inhibitor ONC201 (ONC201) in preventing colorectal cancer in patients with familial adenomatous polyposis (FAP) or a history of multiple polyps. ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

NCT ID: NCT05112822 Not yet recruiting - Clinical trials for Familial Adenomatous Polyposis

Testing Obeticholic Acid (OCA) for Familial Adenomatous Polyposis (FAP)

Start date: March 31, 2022
Phase: Phase 1
Study type: Interventional

This is a trial that intends to evaluate the effect of treatment with the drug obeticholic acid in the treatment of the Familial Adenomatous Polyposis condition.

NCT ID: NCT04531930 Not yet recruiting - Clinical trials for Familial Adenomatous Polyposis

Colorectal Adenoma Canceration in FAP

Start date: October 1, 2020
Phase:
Study type: Observational [Patient Registry]

The current internationally accepted treatment method for familial adenomatous polyposis is prophylactic total colorectal resection combined with endoscopic follow-up. However, total colorectal resection will bring a sharp decline in the quality of life of patients. Therefore, how to improve treatment methods and improve the quality of life for such patients under the premise of medical quality is the current medical focus. This study intends to establish three parallel observation cohorts, namely the surgical treatment group, the intensive colonoscopy treatment group, and the autonomous monitoring group. During the three-year study period, the investigators observed changes in the number of adenomas, carcinogenesis, and medical expenses in each group during the 3-year study period, and compared the groups to determine whether the intensive colonoscopy therapy has the possibility of delaying or replacing preventive surgery.

NCT ID: NCT04454151 Not yet recruiting - Clinical trials for Familial Adenomatous Polyposis

Azithromycin Treatment for Readthrough of APC Gene Stop Codon Mutations in Familial Adenomatous Polyposis

FAP
Start date: August 1, 2020
Phase: Phase 4
Study type: Interventional

Colorectal cancer (CRC) is a leading cause for cancer related mortality in the western world with a lifetime risk of 6%. Etiology is complex, while genetic background significantly affects the risk. Around one third of all genetic disorders as well as most cases of Familial Adenomatous Polyposis (FAP) and a large proportion of all sporadic CRC cases occur as a result of premature nonsense mutations (creating a stop codon) in an individual's adenomatous polyposis coli (APC) gene. Nonsense mutations are single-point alterations in the DNA that prematurely halt the protein translation process, producing a shortened, nonfunctional protein. In many of these cases, if the cell can be 'persuaded' to ignore the premature stop codon signal, the resulting protein may be able to ameliorate or stop the disease. Recently, members of the aminoglycoside family of antibiotics have been found to induce ribosomal read-through of nonsense mutations, leading to expression of a full length, functional protein. Investigators have recently shown that members of the aminoglycoside and macrolide antibiotic families can induce read-through of the nonsense mutations in the APC gene and lead to reduced oncogenic phenotypes in CRC cells and in different mice models. The aim of this project is to determine the ability of the macrolide antibiotic-Azithromycin to induce read-through of the nonsense mutations in the APC gene and to induce expression of a full length, functional APC protein in patients suffering from FAP and to tests its effect on adenoma number and size and on desmoid tumors in these patients. The future goal is to maximize the effect of stop-codon suppressors on APC while minimizing side effects. In this study the investigators will select FAP patients which carry APC nonsense mutations, treat them with Azithromycin PO for 4-6 months and examine colonic and duodenal adenomas as well as abdominal desmoid tumors, that will be documented before during and after treatment. In parallel, investigators will test polyp, adenoma and desmoid tissue samples as well as blood samples from these patients for changes in expression levels of the APC protein and related oncogenic markers. Suppression of nonsense mutations within the APC gene should be of benefit for patients suffering from FAP, attenuated FAP or multiple adenomas and for patients with advanced or diffuse CRC. Furthermore, given the rapid progress being made in the identification of different nonsense mutations in human genes that lead to mostly non-curable disease, the identification of clinically approved compounds that suppress nonsense mutations and that can be administered long-term without significant side effects would open new venues in the treatment of genetic human diseases that arise from pre-mature stop codons in important coding sequences. Immediate goal: establish the ability of Azithromycin to read-through APC nonsense mutation in FAP patients. The read-through effect of Azithromycin will be clinically tested by counting and measuring the number and size of both colonic and duodenal adenomas before and over treatment and by measuring the size of known desmoid tumors. Samples of the adenomas and desmoid tumors will be tested by western blot, immunofluorescence and immunohistochemistry for restoration of APC expression and changes in oncogenic markers. These experiments should be conducted within 6 month. Long term objective: 1. Determine the lowest dose of Azithromycin that can inhibit growth of colonic neoplasia and CRC in patients expressing a truncated APC protein due to nonsense mutations. 2. Examine the ability of a panel of additional macrolide antibiotics to induce APC nonsense mutation suppression using in-vitro methods. Investigators will focus on macrolide antibiotics that are currently in clinical use and are administrated for long terms. These objectives should take around 4 month and will be conducted in parallel.

NCT ID: NCT03061591 Not yet recruiting - Clinical trials for Familial Adenomatous Polyposis

Turmeric Supplementation on Polyp Number and Size in Patients With Familial Adenomatous Polyposis.

Start date: April 2017
Phase: Phase 2
Study type: Interventional

A Phase 2, randomized, double blind, placebo controlled trial for the effectivness of wholistic turmeric supplementation on polyp burden mong patients with Familial Adenomatouse Polyposis (FAP). Fourty Patients will be randomly assigned in a 1:1 ratio to recieve treatment with 8 capsuls (2*4 capsuls/day) of wholistic Turmeric capsules (Pukka herbs) or placebo for six months.