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Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP) (CHIP)

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ClinicalTrials.gov Identifier: NCT00585312
Recruitment Status : Terminated (See termination reason in detailed description.)
First Posted : January 3, 2008
Results First Posted : November 3, 2014
Last Update Posted : November 3, 2014
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Prevention
Condition Adenomatous Polyposis Coli
Interventions Drug: Celecoxib
Drug: Placebo
Enrollment 106
Recruitment Details A total of 305 participants were screened, whereof 106 were randomized into the study, and of whom 101 took at least 1 dose of study drug. The clinical study was conducted in 18 centers across 13 countries: Belgium, Czech Republic, Hong Kong, Hungary, Israel, Italy, Slovakia, South Africa, Spain, Sweden, Ukraine, United Kingdom, and United States.
Pre-assignment Details The randomization was to be stratified by center, age (≥12 years old versus <12 years old), and familial adenomatous polyposis (FAP) phenotype (negative versus positive). The participants were randomized 1:1 to one of the 2 treatments celecoxib or placebo.
Arm/Group Title Celecoxib Placebo
Hide Arm/Group Description Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily. Matching placebo
Period Title: Overall Study
Started 55 [1] 51 [1]
Treated 53 48
Completed 4 7
Not Completed 51 44
Reason Not Completed
Adverse Event             3             0
Lack of Efficacy             6             11
Lost to Follow-up             1             1
Reason not specified             2             0
Withdrawal by Subject             5             1
Study terminated by the sponsor             34             31
[1]
STARTED=Randomized
Arm/Group Title Celecoxib Placebo Total
Hide Arm/Group Description Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily. Matching placebo Total of all reporting groups
Overall Number of Baseline Participants 55 51 106
Hide Baseline Analysis Population Description
The intent-to-treat (ITT) population (N: 106) consisted of all participants who were randomized, with study drug assignment designated according to initial randomization, regardless of whether the participants received any study drug or received a different drug from that to which they were randomized.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 55 participants 51 participants 106 participants
12.6  (2.2) 12.2  (1.8) 12.4  (2.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 55 participants 51 participants 106 participants
Female
29
  52.7%
28
  54.9%
57
  53.8%
Male
26
  47.3%
23
  45.1%
49
  46.2%
1.Primary Outcome
Title Time to Disease Progression
Hide Description

Time to disease progression was defined as the time from randomization to the earliest occurrence of one or more of the following events:

  1. Appearance of ≥20 polyps (>2 mm in size) at any colonoscopy during the study (Polyps); or
  2. Diagnosis of colorectal malignancy (ColMal).
Time Frame 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population (N: 106) consisted of all participants who were randomized and assigned to treatment. Primary outcome measure was met by 7 (Polyp:7,ColMal:0) participants in the Celecoxib group and 13 (13,0) in the placebo group. Study was early terminated due to low enrollment and lower than expected endpoint rate. No analysis was performed.
Arm/Group Title Celecoxib Placebo
Hide Arm/Group Description:
Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily.
Matching placebo
Overall Number of Participants Analyzed 7 13
Mean (Standard Deviation)
Unit of Measure: years
2.2  (1.08) 1.8  (1.30)
2.Secondary Outcome
Title Time to Treatment Failure
Hide Description

Time to treatment failure was defined as time from randomization to the earliest occurrence of one or more of the following:

  • Appearance of ≥20 polyps (>2 mm in size) at any colonoscopy during the study (Polyps), or
  • Diagnosis of colorectal malignancy (ColMal), or
  • Treatment related dropout (DO). The treatment related dropout was defined as insufficient clinical response, progression of disease, death, adverse event, treatment-related laboratory abnormality, subject no longer willing to participate in study, and other reasons that might be related to treatment as determined by treating physicians in a blind fashion before database release.
Time Frame 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population (N: 106) consisted of all participants who were randomized and assigned to treatment. Secondary outcome measure was met by 14 (Polyp:7,ColMal:0,DO:14) participants in the Celecoxib and 14 (13,0,12) in the placebo group. Study was early terminated due to low enrollment and lower than expected endpoint rate. No analysis was performed.
Arm/Group Title Celecoxib Placebo
Hide Arm/Group Description:
Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily.
Matching placebo
Overall Number of Participants Analyzed 14 14
Mean (Standard Deviation)
Unit of Measure: years
2.0  (1.12) 1.7  (1.30)
3.Secondary Outcome
Title Total Number of Colorectal Polyps
Hide Description

Total number of colorectal polyps >2 mm in size, that were detected over Years 1 - 5 cumulatively.

Weighted total number of colorectal polyps over Years 1 – 5 cumulatively was defined as the total number of colorectal polyps >2 mm in size, that were detected over Years 1 - 5, divided by the number of colonoscopies that the participant had during the study.

Time Frame Years 1 - 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population (N: 106) consisted of all participants who were randomized and assigned to a treatment. Study was early terminated due to low enrollment and lower than expected endpoint rate and no analysis was performed.
Arm/Group Title Celecoxib Placebo
Hide Arm/Group Description:
Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily.
Matching placebo
Overall Number of Participants Analyzed 55 51
Mean (Standard Deviation)
Unit of Measure: polyps
Year 1 (N: 27, 30) 3.0  (2.68) 8.1  (7.32)
Year 2 (N: 21, 25) 8.8  (6.63) 13.7  (10.51)
Year 3 (N: 16, 14) 13.4  (11.31) 22.3  (11.74)
Year 4 (N: 8, 7) 18.6  (17.65) 36.4  (22.50)
Year 5 (N: 2, 2) 30.5  (21.92) 46.5  (34.65)
Years 1 - 5 cumulatively (N: 33, 36) 4.3  (3.58) 8.6  (7.12)
4.Secondary Outcome
Title Colorectal Polyp Burden
Hide Description

The polyp burden was defined as the sum of the largest diameters of all polyps (>2 mm in size) over Years 1 - 5 cumulatively.

Weighted colorectal polyp burden over Years 1 – 5 cumulatively was defined as the polyp burden over Years 1 - 5 divided by the number of colonoscopies that the participant had during the study.

Time Frame Years 1 - 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population (N: 106) consisted of all participants who were randomized and assigned to a treatment. Study was early terminated due to low enrollment and lower than expected endpoint rate and no analysis was performed.
Arm/Group Title Celecoxib Placebo
Hide Arm/Group Description:
Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily.
Matching placebo
Overall Number of Participants Analyzed 55 51
Mean (Standard Deviation)
Unit of Measure: mm
Year 1 (N: 27, 30) 4.0  (1.97) 4.2  (2.05)
Year 2 (N: 21, 25) 6.9  (2.28) 8.1  (4.06)
Year 3 (N: 16, 14) 9.6  (3.14) 11.6  (4.05)
Year 4 (N: 8, 7) 12.9  (3.31) 18.7  (5.88)
Year 5 (N: 2, 2) 20.0  (7.07) 20.0  (4.24)
Year 1 - 5 cumulatively (N: 33, 36) 4.1  (1.68) 4.3  (1.61)
Time Frame Events were reported from randomization through and including 30 calendar days after the last administration of the study drug.
Adverse Event Reporting Description The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as nonserious in another participant, or one participant may have experienced both a serious and nonserious event during the study.
 
Arm/Group Title Celecoxib Placebo
Hide Arm/Group Description Celecoxib, approximately 16 mg/kg/day (adjusted for changes in body weight). Maximum dose was 400 mg twice daily. Matching placebo
All-Cause Mortality
Celecoxib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Celecoxib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   3/53 (5.66%)   0/48 (0.00%) 
Infections and infestations     
Periorbital cellulitis * 1  1/53 (1.89%)  0/48 (0.00%) 
Pneumonia * 1  1/53 (1.89%)  0/48 (0.00%) 
Psychiatric disorders     
Depression * 1  1/53 (1.89%)  0/48 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 16.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Celecoxib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   36/53 (67.92%)   30/48 (62.50%) 
Gastrointestinal disorders     
Abdominal discomfort * 1  9/53 (16.98%)  4/48 (8.33%) 
Abdominal pain * 1  9/53 (16.98%)  10/48 (20.83%) 
Abdominal pain upper * 1  4/53 (7.55%)  3/48 (6.25%) 
Diarrhoea * 1  6/53 (11.32%)  4/48 (8.33%) 
Nausea * 1  8/53 (15.09%)  8/48 (16.67%) 
Rectal haemorrhage * 1  2/53 (3.77%)  3/48 (6.25%) 
Vomiting * 1  9/53 (16.98%)  9/48 (18.75%) 
General disorders     
Chest pain * 1  4/53 (7.55%)  1/48 (2.08%) 
Fatigue * 1  6/53 (11.32%)  4/48 (8.33%) 
Immune system disorders     
Seasonal allergy * 1  2/53 (3.77%)  5/48 (10.42%) 
Infections and infestations     
Ear infection * 1  4/53 (7.55%)  2/48 (4.17%) 
Gastroenteritis * 1  3/53 (5.66%)  2/48 (4.17%) 
Gastroenteritis viral * 1  3/53 (5.66%)  1/48 (2.08%) 
Influenza * 1  6/53 (11.32%)  1/48 (2.08%) 
Nasopharyngitis * 1  6/53 (11.32%)  4/48 (8.33%) 
Upper respiratory tract infection * 1  4/53 (7.55%)  9/48 (18.75%) 
Viral infection * 1  0/53 (0.00%)  4/48 (8.33%) 
Investigations     
Albumin urine present * 1  3/53 (5.66%)  0/48 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia * 1  3/53 (5.66%)  2/48 (4.17%) 
Pain in extremity * 1  2/53 (3.77%)  5/48 (10.42%) 
Nervous system disorders     
Headache * 1  16/53 (30.19%)  14/48 (29.17%) 
Migraine * 1  2/53 (3.77%)  4/48 (8.33%) 
Respiratory, thoracic and mediastinal disorders     
Cough * 1  8/53 (15.09%)  6/48 (12.50%) 
Dyspnoea * 1  3/53 (5.66%)  1/48 (2.08%) 
Epistaxis * 1  0/53 (0.00%)  3/48 (6.25%) 
Oropharyngeal pain * 1  6/53 (11.32%)  5/48 (10.42%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA 16.1
The study was early terminated and, due to the low number of participants, no efficacy analysis was performed. Only descriptive statistics was performed.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Results Point of Contact
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
Phone: 1-800-718-1021
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00585312     History of Changes
Obsolete Identifiers: NCT00393016, NCT00534040
Other Study ID Numbers: A3191193
First Submitted: January 1, 2008
First Posted: January 3, 2008
Results First Submitted: October 28, 2014
Results First Posted: November 3, 2014
Last Update Posted: November 3, 2014