Treatment for Familial Adenomatous Polyposis (FAP)

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Johns Hopkins Hospital, Baltimore, MD
Familial Adenomatous Polyposis (FAP)+3 More
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a new treatment for duodenal adenomas is safe and effective.

See full description

Eligible Conditions

  • Familial Adenomatous Polyposis (FAP)
  • Duodenal Adenomas

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Treatment will improve 3 primary outcomes and 7 secondary outcomes in patients with Familial Adenomatous Polyposis (FAP). Measurement will happen over the course of 1 year.

1 year
Complete eradication rate of DAs
5 years
Median number of CryoBalloon treatments to complete eradication.
Progression rate to high grade dysplasia or duodenal cancer
Safety of cryoBalloon ablation in treating non-ampullary non-polypoid duodenal adenomas (DAs) as assessed by the incidence of adverse events in all treated patients
Safety of cryoBalloon ablation in treating non-ampullary non-polypoid duodenal adenomas (DAs) as assessed by the incidence of adverse events in all treatment procedures
Technical failure rate
Time to complete eradication of DAs in each patient
Time to complete eradication of each treated DA lesion
Year 5
Change in Spigelman class score
Percent change in the treated duodenal adenoma size

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Familial Adenomatous Polyposis (FAP)
1 of 2
Sporadic duodenal adenomas
1 of 2
Active Control

This trial requires 50 total participants across 2 different treatment groups

This trial involves 2 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

Familial Adenomatous Polyposis (FAP)
Device
Individuals with duodenal adenomas (DAs) and FAP with Spigelman class 2,3 or 4, treated with cryoballoon ablation (intervention)
Sporadic duodenal adenomas
Device
Individuals with at least 1 sporadic duodenal adenoma (DA) between 1-5 cm in maximum diameter, treated with cryoballoon ablation (intervention)

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, 1 year, 2 years, 3 years, 4 years, 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline, 1 year, 2 years, 3 years, 4 years, 5 years for reporting.

Closest Location

Johns Hopkins Hospital - Baltimore, MD

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
You have a history of prior EMR or saline-assisted polypectomy. show original
You have a duodenal adenoma between 1 and 5 cm in width. show original
You have FAP with Spigelman class 2, 3 or 4. show original
Polyp characteristics: Non-polypoid lesions Paris 2A and 2B, or Sessile adenomas, occupying no more than 50% circumference of duodenum, and no more than 3 duodenal folds. show original
Individuals must be considered high risk for surgery or endoscopic resection, due to complication risk, or declined standard therapies. show original

Patient Q&A Section

What is adenoma?

"Adenomatous polyps occur in the gastrointestinal tract and may develop from other tumors. They are usually nonpainful and are found in the colon. The majority of colorectal polyps, and some adenomas, are benign.\n" - Anonymous Online Contributor

Unverified Answer

What causes adenoma?

"The cause of adenomas is not well understood. It is likely that the process involves combination of a number of genetic and environmental factors. Some viruses have been implicated in the cause of adenomas." - Anonymous Online Contributor

Unverified Answer

How many people get adenoma a year in the United States?

"In the United States, about 8.7 million procedures are performed annually in non-surgical settings for adenoma or hyperplasia. Most are related to adenoma or hyperplasia of the colorectum as the patient is most likely to seek medical care following a screening colonoscopy. Many more procedures are performed for adenomatous or hyperplastic polyps in the esophagus than in the colon--a condition which, if not treated, seldom has symptoms. The yearly number of new diagnoses of both adenoma and hyperplasia of the esophagus, larynx and stomach may thus be about 2 to 8 million." - Anonymous Online Contributor

Unverified Answer

What are common treatments for adenoma?

"Adenoma can produce a number of symptoms, but no one adenoma is the cause of all symptoms. The treatment of adenoma depends on the cause of the symptoms. Adenoma of the bladder is usually dealt with by cystoscopy and sometimes by surgical excision. A number of treatments may be appropriate for a patient with symptoms of adenocarcinoma, but often radiotherapy is the mainstay. Adenomatosis can produce significant symptoms, and may require surgical excision. A minority of adenomas produce symptoms from a mass. In these cases, radiotherapy may be required. A small proportion of patients may require hormonal or other treatment." - Anonymous Online Contributor

Unverified Answer

What are the signs of adenoma?

"In practice doctors are usually unsure of the significance of many of the signs. Although not a sign of adenoma, the possibility of cancer is raised by changes in bowel habit. The most important signs of cancer are weight loss, blood in the stool, and pain when swallowing. The signs of bleeding do not necessarily imply cancer, as may be the case in ulcerative colitis. Pain may be caused by a peptic ulcer. Most of the time physicians think the only possibility is ulcerative colitis. However, 10% of patients with severe ulcerative colitis may have adenoma attached to the colonic mucosa of which the endoscopic appearance would mimic a malignant lesion." - Anonymous Online Contributor

Unverified Answer

Does treatment improve quality of life for those with adenoma?

"Data from a recent study suggest that for those at high risk for colorectal neoplasia, careful medical evaluation and careful decision making in the context of one's values can improve quality of life. The treatment regimen did not have an effect on the quality of life of individuals who had adenomas. Further research should investigate other aspects of colorectal neoplasia treatment, including the impact of the disease and lifestyle interventions." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of adenoma?

"Adenomas tend to be a consequence of an underlying cause. The risk of an adenoma is much higher with cigarette smoking and less with physical activity." - Anonymous Online Contributor

Unverified Answer

Does adenoma run in families?

"Results from a recent clinical trial indicates that familial adenomatous polyposis (FAP) accounts for 2.0% of [colorectal cancer](https://www.withpower.com/clinical-trials/colorectal-cancer) (CRC) cases in Japan, while it is much lower in the western world. Results from a recent clinical trial also support that sporadic cases of CRC, especially advanced CRC (Stage IIA, IIB, or IIIC cancers) are caused by CRCs in an underlying FAP. The underlying FAP of CRC cases in Japan (1:27,000 cases) are mostly autosomal dominant ones whereas the underlying FAP of CRC cases in the West are largely autosomal recessive (1:600,000 cases). We speculate that CRC in Japan may be a phenotype associated with sporadic CRC in the West." - Anonymous Online Contributor

Unverified Answer

How serious can adenoma be?

"Patients with AA and AP adenoma are more likely to be non-Hispanic than those with AP AA. However, the risk of cancer progression is still elevated in the group with AA adenoma, but not in the AP group; this difference cannot be explained by ethnicity. The authors therefore do not know whether AP patients with AA adenoma are actually at increased risk for cancer progression." - Anonymous Online Contributor

Unverified Answer

What does treatment usually treat?

"Most benign adenomas warrant surgical removal before they cause a disease complication. The prevalence of malignancy is less than 1% for patients with small carcinomas and less than 5% for patients with adenomas larger than 2 cm in diameter. Patients with large lesions that cause symptoms should undergo a biopsy and proceed with treatment as needed before the adenoma is removed because the incidence of malignancy is so low." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of treatment?

"Treatment with sorafenib [vs. placebo] did not result in increased severity of side effects when compared to placebo in patients with advanced solid tumors. Treatment of patients with advanced solid tumors with sorafenib has been well tolerated despite a high incidence of side effects. Sorafenib-treated patients receiving interstitial radiation therapy for brain metastases experienced increased toxicity rates compared to those treated with radiation therapy alone. There appears to be no increased risk of malignancy associated with sorafenib therapy. The incidence and frequency of grade ≥ 2 diarrhea or nausea/vomiting increased significantly with sorafenib and interstitial brachytherapy but not with sorafenib alone." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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