NCT02961374

Erlotinib Hydrochloride in Reducing Duodenal Polyp Burden in Patients With Familial Adenomatous Polyposis at Risk of Developing Colon Cancer

Phase II Trial of Weekly Erlotinib Dosing to Reduce Duodenal Polyp Burden Associated With Familial Adenomatous Polyposis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years – 69 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies the side effects of erlotinib hydrochloride and how well it works in reducing duodenal polyp burden in patients with familial adenomatous polyposis at risk of developing colon cancer. Erlotinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVES: I. To assess the mean percent change in duodenal polyp burden (sum of diameters from all polyps) from baseline to 6 months post-intervention for familial adenomatous polyposis (FAP) subjects receiving weekly erlotinib hydrochloride (erlotinib). II. To assess the grade 2/3 adverse event rate in this population and compare it to historical data. SECONDARY OBJECTIVES: I. To evaluate all adverse events at least possibly attributed to weekly erlotinib. II. To assess the absolute and percent change in duodenal polyp number from baseline to 6 months. III. To assess the absolute and percent changes in lower gastrointestinal polyp burden and number for the subset of participants with ileal pouch anal anastomosis (IPAA) or ileo-rectal anastomosis with rectal stump. IV. To assess the absolute and percent change in desmoid tumor size in participants who have baseline and follow up computed tomography (CT)s performed as part of their standard of care. CORRELATIVE OBJECTIVES: I. Gene expression profiles in duodenal adenomas and uninvolved tissue will be compared between baseline and endpoint samples using negative binomial statistics (DESeq2). II. Identify differentially expressed genes between duodenal polyps and uninvolved tissue at endpoint compared to baseline. III. Evaluate the effect of weekly erlotinib on EGFR and Wnt target gene expression in duodenal adenomas. IV. Evaluate the effect of weekly erlotinib on immune response signaling in duodenal adenomas and uninvolved tissue. OUTLINE: Patients receive erlotinib hydrochloride orally (PO) once weekly. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days.

Conditions

• Attenuated Familial Adenomatous Polyposis
• Familial Adenomatous Polyposis

Interventions

Type	Name	Description
DRUG	Erlotinib	Given PO
DRUG	Erlotinib Hydrochloride	Given PO

Timeline

• Start date: 2017-10-27
• Primary completion: 2020-02-23
• Completion: 2021-09-27
• First posted: 2016-11-11
• Last updated: 2022-07-26
• Results posted: 2021-09-22

Locations

8 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study