Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)

This study has been withdrawn prior to enrollment.
Information provided by:
Cancer Prevention Pharmaceuticals, Inc. Identifier:
First received: November 19, 2010
Last updated: May 27, 2011
Last verified: May 2011

The purpose of this phase III study is to evaluate the safety and efficacy of the combination of eflornithine and sulindac compared to single agent sulindac or eflornithine in reducing the number of polyps in patients with familial adenomatous polyposis (FAP).

Condition Intervention Phase
Familial Adenomatous Polyposis
Drug: Eflornithine plus Sulindac
Drug: Eflornithine plus Placebo
Drug: Sulindac plus Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized Phase III Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)

Resource links provided by NLM:

Further study details as provided by Cancer Prevention Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Efficacy of Eflornithine plus Sulindac compared to Eflornithine alone and Sulindac alone determined by change in the number of polyps 2 mm or greater in a defined focal area of the rectum or pouch at baseline and after completion of the study treatment. [ Time Frame: 6 months from the start of treatment. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in number of polyps 2 mm or greater in the distal 10 cm of rectum or pouch. [ Time Frame: 6 months from the start of treatment. ] [ Designated as safety issue: No ]
  • Qualitative change in overall colon/rectum/pouch polyp burden (number and size). [ Time Frame: 6 months from the start of treatment. ] [ Designated as safety issue: No ]
  • Presence of high grade dysplasia or villous adenoma in any polyp resected. [ Time Frame: 6 months from the start of treatment. ] [ Designated as safety issue: No ]

Estimated Enrollment: 140
Study Start Date: March 2011
Estimated Study Completion Date: June 2013
Estimated Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eflornithine plus Sulindac
Eflornithine 500 mg and Sulindac 150 mg
Drug: Eflornithine plus Sulindac
Eflornithine, 250 mg tablet, two tablets (500 mg) orally once a day; Sulindac, 150 mg tablet, one tablet orally once a day
Other Name: DFMO
Active Comparator: Elfornithine plus Placebo
Eflornithine 500 mg and Placebo
Drug: Eflornithine plus Placebo
Eflornithine, 250 mg tablet, two tablets (500 mg) orally once a day; Placebo, one tablet orally once a day
Other Name: DFMO
Active Comparator: Sulindac plus Placebo
Sulindac 150 mg and Placebo
Drug: Sulindac plus Placebo
Sulindac, 150 mg tablet, one tablet orally once a day; Placebo, two tablets orally once a day


Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of phenotypic Familial Adenomatous Polyposis (FAP) of the colorectum based on meeting the criteria in one of two groups: Group 1-Greater than 100 adenomatous colorectal polyps prior to age 40. Group 2-Greater than 10 adenomatous polyps and age <40 or greater than 25 polyps and age >40; combined with a dominant family history or genotype: More than 100 polyps in a first-degree relative; More than 25 polyps in 2 relatives in 2 generations, including a first-degree family member; Genetic diagnosis in a relative; Genetic diagnosis by in vitro synthesized truncated protein or similar assay.
  • No colorectal surgery or prior colon surgery for polyposis at least 1 year prior (total abdominal colectomy with ileal-rectal anastomosis, or total proctocolectomy with ilea pouch-anal reconstruction.
  • Baseline endoscopy

    1. If no prior colorectal surgery, at least 3 polyps in a cluster each ≥ 2 mm in diameter; or
    2. If rectum is in situ and to be assessed, baseline rectal segment endoscopy documenting 3 or more rectal polyps each at least 2 mm in diameter in a defined cluster and/or at least 6 polyps, ≥ 2 mm in diameter, in the distal 10 cm of rectum
    3. If ileal pouch neo-rectum is in place, 3 or more pouch polyps in a cluster ≥ 2 mm in diameter, or at least 6 polyps, ≥ 2 mm in diameter, in the distal 10 cm of pouch.
    4. Clinical/pathological grading of duodenal polyps will utilize the Spigelman Classification.
  • Hematopoietic: no significant hematologic dysfunction; WBC ≥3,000/mm3; platelet count ≥100,000/mm3; hemoglobin ≥10g/dL; no known or prior clinical coagulopathy.
  • Hepatic: bilirubin ≤ 1.5 times ULN; AST and ALT ≤ 1.5 times ULN; Alkaline phosphatase ≤ 1.5 times ULN.
  • Renal: No significant renal dysfunction; creatinine ≤ 1.5 times ULN.
  • Hearing: no clinically significant hearing loss that affects everyday life.
  • Not pregnant or nursing.
  • Negative serum pregnancy test if female of child-bearing potential.
  • Absence of gross blood in stool.
  • Fertile patients must use effective contraception.
  • Stool occult blood either negative or minimal (1+).
  • No prior hypersensitivity to cyclooxygenase-2 inhibitors, sulfonamides, NSAIDs, or salicylates; no NSAID associated symptoms of gastritis.
  • No discrete gastric or duodenal ulcer greater than 5 mm within the past year except Helicobacter pylori-related peptic ulcer disease treated successfully with antibiotics (as documented by an endoscopy.
  • No invasive malignancy within the past 5 years except stage I or II colon or rectal cancer or resected nonmelanomatous skin cancer.
  • No other significant medical or psychiatric problems that would preclude study participation.
  • No chronic adrenocorticosteroids.
  • No prior pelvic irradiation.
  • At least 3 months since prior investigational agents.
  • Patients may not be receiving or plan to receive corticosteroids.
  • Concomitant NSAID use outside this study may not exceed 4 days per month.
  • Use of 81 mg daily aspirin or 650 mg aspirin not more than once a week.
  • No concurrent warfarin, fluconazole, or lithium.
  • Must be willing and able to sign informed consent.

Exclusion Criteria:

  • High Risk for cardiovascular disease including clinical diabetes mellitus (Type I or II) requiring glycemic medications; Prior personal history of cardiovascular disease or, two or more of the following - hypertension or use of anti-hypertensive medications, hyperlipidemia or use of lipid-lowering medications or current smoker.
  • Hearing loss that affects everyday life and or for which a hearing aid is required.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01245816

Sponsors and Collaborators
Cancer Prevention Pharmaceuticals, Inc.
Study Director: Alfred M. Cohen, M.D. Cancer Prevention Pharmaceuticals, Inc.
  More Information

No publications provided

Responsible Party: Jeffrey Jacob, CEO, Cancer Prevention Pharmaceuticals, Inc. Identifier: NCT01245816     History of Changes
Other Study ID Numbers: CPP FAP-301
Study First Received: November 19, 2010
Last Updated: May 27, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Cancer Prevention Pharmaceuticals, Inc.:
colon polyps

Additional relevant MeSH terms:
Adenomatous Polyposis Coli
Adenomatous Polyps
Colonic Diseases
Colonic Neoplasms
Colorectal Neoplasms
Digestive System Diseases
Digestive System Neoplasms
Gastrointestinal Diseases
Gastrointestinal Neoplasms
Genetic Diseases, Inborn
Intestinal Diseases
Intestinal Neoplasms
Intestinal Polyposis
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Glandular and Epithelial
Neoplastic Syndromes, Hereditary
Analgesics, Non-Narcotic
Anti-Infective Agents
Anti-Inflammatory Agents
Anti-Inflammatory Agents, Non-Steroidal
Antineoplastic Agents
Antiparasitic Agents
Antiprotozoal Agents
Antirheumatic Agents processed this record on March 03, 2015