Familial Adenomatous PolyposisPhase 3NCT01245816
A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
A Phase 3 study testing Eflornithine plus Sulindac in people with Familial Adenomatous Polyposis, run by Cancer Prevention Pharmaceuticals, Inc..
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Familial Adenomatous Polyposis
- What is being tested?
- Eflornithine plus Sulindac
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Mar 2011to Jun 2013
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
- If no prior colorectal surgery, at least 3 polyps in a cluster each ≥ 2 mm in diameter; or
- 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
- 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.
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- 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.
Who cannot join
- 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.
About this study
Official title: A Randomized Phase III Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
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).
- Conditions
- Familial Adenomatous Polyposis
- Being tested
- Eflornithine plus Sulindac, Eflornithine plus Placebo, Sulindac plus Placebo
- Keywords
- colon polyps
- Medical subject headings
- Adenomatous Polyposis Coli, Colonic Polyps
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Eflornithine plus SulindacDrug
Also called DFMO
Eflornithine, 250 mg tablet, two tablets (500 mg) orally once a day; Sulindac, 150 mg tablet, one tablet orally once a day
Eflornithine plus PlaceboDrug
Also called DFMO
Eflornithine, 250 mg tablet, two tablets (500 mg) orally once a day; Placebo, one tablet orally once a day
Sulindac plus PlaceboDrug
Sulindac, 150 mg tablet, one tablet orally once a day; Placebo, two tablets orally once a day
| Study group | Role | What they receive |
|---|---|---|
| Eflornithine plus Sulindac | Experimental | Eflornithine 500 mg and Sulindac 150 mg Receives: Drug: Eflornithine plus Sulindac |
| Elfornithine plus Placebo | Active comparator | Eflornithine 500 mg and Placebo Receives: Drug: Eflornithine plus Placebo |
| Sulindac plus Placebo | Active comparator | Sulindac 150 mg and Placebo Receives: Drug: Sulindac plus Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months from the start of treatment.
Secondary outcomes (3)
Change in number of polyps 2 mm or greater in the distal 10 cm of rectum or pouch.
Measured: 6 months from the start of treatment.
Qualitative change in overall colon/rectum/pouch polyp burden (number and size).
Measured: 6 months from the start of treatment.
Presence of high grade dysplasia or villous adenoma in any polyp resected.
Measured: 6 months from the start of treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2010 (estimated)
- Study startMar 2011
- Primary completionMay 2013 (estimated)
- Study completionJun 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Adenomatous PolyposisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cancer Prevention Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Alfred M. Cohen, M.D., Study director, Cancer Prevention Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01245816
- Sponsor's protocol ID
- CPP FAP-301
- Status last verified
- Apr 2015
- First submitted
- 19 Nov 2010
- Last updated
- 24 Apr 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase III Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
- ClinicalTrials.gov ID
- NCT01245816
- Acronym / sponsor ID
- CPP FAP-301
- Conditions
- Familial Adenomatous Polyposis
- Keywords
- colon polyps
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Cancer Prevention Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2011
- Primary completion
- May 2013 (estimated)
- Study completion
- Jun 2013 (estimated)
- First posted
- 23 Nov 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Eflornithine plus Sulindac | Experimental | Drug: Eflornithine plus SulindacEflornithine 500 mg and Sulindac 150 mg |
| Elfornithine plus Placebo | Active comparator | Drug: Eflornithine plus PlaceboEflornithine 500 mg and Placebo |
| Sulindac plus Placebo | Active comparator | Drug: Sulindac plus PlaceboSulindac 150 mg and Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (28 listed)
- 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
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Eflornithine plus SulindacAlso: DFMOEflornithine, 250 mg tablet, two tablets (500 mg) orally once a day; Sulindac, 150 mg tablet, one tablet orally once a day | Drug | Eflornithine plus Sulindac |
| Eflornithine plus PlaceboAlso: DFMOEflornithine, 250 mg tablet, two tablets (500 mg) orally once a day; Placebo, one tablet orally once a day | Drug | Elfornithine plus Placebo |
| Sulindac plus PlaceboSulindac, 150 mg tablet, one tablet orally once a day; Placebo, two tablets orally once a day | Drug | Sulindac plus Placebo |
Primary outcome
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.
3 secondary outcomes
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.
Qualitative change in overall colon/rectum/pouch polyp burden (number and size).
Time frame: 6 months from the start of treatment.
Presence of high grade dysplasia or villous adenoma in any polyp resected.
Time frame: 6 months from the start of treatment.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alfred M. Cohen, M.D. study director
Cancer Prevention Pharmaceuticals, Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eflornithine plus Sulindac in people with Familial Adenomatous Polyposis, run by Cancer Prevention Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 28 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 24 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2015.