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Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)

C

Cancer Prevention Pharmaceuticals

Status and phase

Completed
Phase 3

Conditions

Familial Adenomatous Polyposis

Treatments

Drug: Eflornithine Placebo
Drug: Sulindac placebo
Drug: Sulindac 150 MG
Drug: Eflornithine

Study type

Interventional

Funder types

Industry

Identifiers

NCT01483144
CPP-FAP-310

Details and patient eligibility

About

The purpose of this randomized, double-blind, Phase III trial is to determine if the combination of eflornithine plus sulindac is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of any FAP-related event. This includes: 1) FAP related disease progression indicating the need for excisional intervention involving the colon, rectum, pouch, duodenum and/or 2) clinically important events which includes progression to more advanced duodenal polyposis, cancer or death.

Enrollment

171 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Diagnosis of phenotypic classical FAP with disease involvement of the duodenum and/or colon/rectum/pouch.

    1. Genotype: Adenomatous polyposis coli (APC) mutation (with or without family history) required
    2. Classical FAP Phenotype: 100's to 1,000's of colorectal adenomatous polyps, usually appearing in teenage years
  • Upper gastrointestinal (UGI) endoscopy/ lower gastrointestinal (LGI) endoscopy (proctoscopy/colonoscopy) performed within 30 days of randomization.

  • Patients with an intact colon/rectum, except for clinical polyposis, and prophylactic surgery is being considered as a stratification site.

  • Rectal/pouch polyposis as a stratification site as follows:

    1. At least three years since colectomy with ileorectal anastamosis (IRA)/proctocolectomy with pouch, and demonstrating polyposis as defined by Stage 1, 2, 3, of the proposed InSiGHT 2011 Staging System (Appendix B) and summarized as follows:

      Stage 1: 10-25 polyps, all < 5 mm Stage 2: 10-25 polyps, at least one > 1 cm Stage 3: >25 polyps amenable to complete removal, or any incompletely removed sessile polyp, or any evidence of high grade dysplasia, even if completely removed. [Note: For staging purposes only.]

    2. For all subjects, any rectal/pouch polyps > 5 mm must be excised at "baseline".

  • Duodenal polyposis as a stratification site; one or more of the following:

    1. Current Spigelman Stage 3 or 4.
    2. Prior surgical endoscopic intervention within the past six months for Spigelman Stage 3 or 4 that may have been down staged to Spigelman Stage 1 or 2.
  • Hematopoietic Status (within 30 days prior to randomization):

    1. No significant hematologic abnormalities
    2. White blood cell count (WBC) at least 3,000/mm3
    3. Platelet count at least 100,000/mm3
    4. Hemoglobin at least 10.0 g/dL
    5. No history of clinical coagulopathy
  • Hepatic Status (within 30 days prior to randomization):

    1. Bilirubin no greater than 1.5 times ULN
    2. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) no greater than 1.5 times ULN
    3. Alkaline phosphatase no greater than 1.5 times ULN
  • Renal Status (within 30 days prior to randomization):

    a) Creatinine no greater than 1.5 times ULN

  • Hearing:

    a) No clinically significant hearing loss, defined in Section 6.2, number 9.

  • If female, neither pregnant nor lactating.

  • Negative pregnancy test if female of child-bearing potential. Fertile patients must use effective contraception*.

  • Absence of gross blood in stool; red blood on toilet paper only acceptable.

  • No discrete gastric or duodenal ulcer greater than 5 mm within the past year except Helicobacter pylori-related peptic ulcer disease treated with antibiotics.

  • No invasive malignancy within the past 5 years except resected non-melanomatous skin cancer, papillary thyroid cancer, or precancerous cervical dysplasia.

  • No other significant medical or psychiatric problems that would preclude study participation or interfere with capacity to give informed consent.

  • Use of 81-100 mg daily aspirin or up to 700 mg aspirin not more than once a week are eligible.

  • No concurrent warfarin, fluconazole, lithium, Pradaxa® or other direct thrombin inhibitors, Plavix®, cyclosporine, other NSAIDs (such as ibuprofen, aspirin, diflunisal), diuretics (furosemide and thiazides), dimethylsulfoxide (DMSO), methotrexate, probenecid, propoxyphene hydrochloride, Tylenol® (acetaminophen) preparations containing aspirin or cytotoxic chemotherapy drugs.

  • Willingness to forego concurrent use of supplements containing omega-3 fatty acids, corticosteroids, non-steroidal anti-inflammatory drugs or other FAP directed drug therapy.

  • Able to provide informed consent and follow protocol requirements.

Exclusion criteria

  • Prior pelvic irradiation.

  • Patients receiving oral corticosteroids within 30 days of enrollment.

  • Treatment with other investigational agents in the prior 4 weeks.

  • Use of other non-steroidal anti-inflammatory drugs (such as ibuprofen) exceeding 4 days per month, in the prior 6 weeks.

  • Regular use of aspirin in excess of 700 mg per week.

  • Treatment with other FAP directed drug therapy (including sulindac or celecoxib, fish oil) within 12 weeks of study enrollment.

  • Hypersensitivity to cyclooxygenase-2 inhibitors, sulfonamides, NSAIDs, or salicylates; NSAID associated symptoms of gastritis.

  • Patients must not have cardiovascular disease risk factors as defined below:

    • Uncontrolled high blood pressure (systolic blood pressure > 150 mm Hg
    • Unstable angina
    • History of documented myocardial infarction or cerebrovascular accident
    • New York Heart Association Class III or IV heart failure
    • Known uncontrolled hyperlipidemia defined as LDL-C >= 190 mg/dL or triglycerides >= 500 mg/dL
  • Patients with significant hearing loss are not eligible for study participation defined as hearing loss that affects everyday life and/or for which a hearing aid is required.

  • Colon/rectum/pouch with high grade dysplasia or cancer on biopsy or a large polyp (>1 cm) not amenable to complete removal.

  • Duodenal cancer on biopsy.

  • Intra-abdominal desmoid disease, stage III or IV

  • Inability to provide informed consent.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Triple Blind

171 participants in 3 patient groups

Eflornithine plus Sulindac
Experimental group
Description:
Eflornithine 750 mg and Sulindac 150 mg
Treatment:
Drug: Sulindac 150 MG
Drug: Eflornithine
Eflornithine plus Sulindac Placebo
Active Comparator group
Description:
Eflornithine 750 mg and Placebo
Treatment:
Drug: Sulindac placebo
Drug: Eflornithine
Sulindac plus Eflornithine Placebo
Active Comparator group
Description:
Sulindac 150 mg and Placebo
Treatment:
Drug: Sulindac 150 MG
Drug: Eflornithine Placebo

Trial documents
2

Trial contacts and locations

17

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Data sourced from clinicaltrials.gov

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