AzTrials
AzTrials

Ulcerative ColitisPhase 3NCT00537316

Efficacy & Safety of Infliximab Monotherapy Vs Combination Therapy Vs AZA Monotherapy in Ulcerative Colitis (Part 1) Maintenance Vs Intermittent Therapy for Maintaining Remission (Part 2)(Study P04807)

A Phase 3 study testing Infliximab (IFX) and Azathioprine (AZA) in people with Ulcerative Colitis, run by Merck Sharp & Dohme LLC.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

Part 1 of this study is a 3-arm, randomized, active-controlled, parallel-group, multicenter, double-blind, double-dummy, 16-week study to compare the efficacy and safety of infliximab (IFX), as monotherapy or in combination with azathioprine (AZA) versus AZA monotherapy in adults with moderate to severe active… Read more about this study

Who is it for?
Ages 21 years and older
Conditions the study is for
Ulcerative Colitis
What is being tested?
Infliximab (IFX) and Azathioprine (AZA)
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
242 participants
Where
No sites listed yet
Study dates
Jul 2007to Feb 2010
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 21 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • must be >=21 years of age at the time of informed consent, of either sex, and of any race;
  • must have endoscopic evidence of UC, as determined by sigmoidoscopy, within 14 days prior to Baseline;
  • must have a total Mayo score of 6 to 12 points at Baseline;
  • must have responded inadequately to corticosteroid treatment (ie, the last or current UC flare did not respond adequately to a standard course of corticosteroids) with or without 5 aminosalicylic acid (5-ASA);
  • must be off corticosteroids or on a stable dose of corticosteroid for at least 2 weeks prior to enrollment. The maximal daily dose of corticosteroid at Baseline must not exceed the equivalent of 30 mg of prednisone;
  • must be naïve to infliximab and other tumor necrosis factor-alpha (TNF-α) antagonists;
Show all 21Show fewer
  • must be either naïve to AZA/6-MP or have not received AZA/6-MP for at least 3 months before enrollment in the study;
  • considered eligible according to the following tuberculosis (TB) screening criteria:
  • have no history of latent or active TB prior to Screening;
  • have no signs or symptoms suggestive of active TB upon medical history and/or physical examination;
  • have had no recent close contact with a person with active TB or, if there has been such contact, will be referred to a physician specializing in TB to undergo additional evaluation and, if warranted, receive appropriate treatment for latent TB prior to or simultaneously with the first administration of IFX;
  • within 1 month prior to the first administration of infliximab, either have negative tuberculin skin test OR have a newly identified positive tuberculin test during Screening in which active TB has been ruled out, and for which appropriate treatment for latent TB has been initiated either prior to or simultaneously with the first administration of IFX.
  • must have a chest X-ray (posterior-anterior and lateral views), taken within 3 months prior to the first administration of study agent and read by a qualified radiologist, with no evidence of current active TB or old active TB;
  • have had UC for more than 10 years should have had a full colonoscopy within 2 years prior to Screening for the surveillance of dysplasia;
  • screening and Baseline clinical laboratory tests (complete blood count [CBC] and blood chemistries) must be within predetermined parameters
  • had been on antibiotics for the treatment of UC (eg, ciprofloxacin and metronidazole) must have been discontinued from them at least 3 weeks prior to Screening;
  • must be free of any clinically significant condition or situation, other than UC that, in the opinion of the investigator, would interfere with the study evaluations or optimal participation in the study;
  • willing and able to adhere to the study visit schedule and other protocol requirements;
  • capable of providing written informed consent, which must be obtained prior to conducting any protocol-specified procedures;
  • women of child-bearing potential and all men must agree to use a medically accepted method of contraception prior to screening, while receiving protocol-specified medication, and for 6 months after stopping the medication. Acceptable methods of contraception include condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide, medically prescribed intrauterine device (IUD), oral or injectable hormonal contraceptive, and surgical sterilization (eg, hysterectomy or tubal ligation). Women of child-bearing potential who are not currently sexually active must agree to use a medically accepted method of contraception should they become sexually active while participating in the study;
  • female participants of childbearing potential must have a negative serum pregnancy test (beta-human chorionic gonadotropin) at Screening and a negative urine pregnancy test at Baseline.

Who cannot join

  • have severe extensive colitis as evidenced by:
  • investigator judgment that the participant is likely to require colectomy within 12 weeks of Baseline
  • OR
  • at least 4 of these symptoms at Screening or Baseline visits, as follows:
  • diarrhea with >=6 bowel movements/day with macroscopic blood in stool;
  • focal severe or rebound abdominal tenderness;
Show all 37Show fewer
  • persistent fever (>=37.5 degrees C) for at least 3 days prior to baseline;
  • tachycardia (>100 beats/minute);
  • hemoglobin <8.5 g/dL (5.3 mM/L).
  • require, or are required within the 2 months prior to baseline, surgery for active gastrointestinal bleeding, peritonitis, intestinal obstruction, or intra-abdominal or pancreatic abscess requiring surgical drainage or other conditions possibly confounding the evaluation of disease activity;
  • have severe, fixed symptomatic stenosis of the large or small intestine;
  • have current evidence of colonic obstruction or history within the 6 months prior to baseline, confirmed with objective radiographic or endoscopic evidence of a stricture with resulting obstruction (dilation of the colon proximal to the stricture on barium radiograph or an inability to traverse the stricture at endoscopy);
  • have a history of colonic mucosal dysplasia;
  • presence on screening endoscopy of adenomatous colonic polyps, if not removed prior to study entry, or history of adenomatous colonic polyps that were not removed;
  • have the presence of a stoma;
  • have a history of extensive colonic resection that would prevent adequate evaluation of clinical disease activity (eg, less than 30 cm of colon remaining);
  • have had a positive stool culture for enteric pathogens, pathogenic ova or parasites within 4 months prior to Baseline unless participant has received treatment and had a negative stool examination 1 week or longer after the end of treatment;
  • have a concomitant diagnosis of congestive heart failure (CHF), including medically controlled asymptomatic subjects;
  • have had serious infections (eg, active hepatitis, pneumonia, or pyelonephritis) within 2 months of Screening. Less serious infections (such as acute upper respiratory tract infection [colds] or a simple urinary tract infection) need not be considered as an exclusion at the discretion of the investigator;
  • have had a nontuberculous mycobacterial infection or opportunistic infection (eg, cytomegalovirus, Pneumocystis carinii, aspergillosis) within 6 months prior to Screening;
  • have a known infection with human immunodeficiency virus (HIV) and/or hepatitis B or hepatitis C;
  • have a history of a known allergy to murine proteins or allergy/sensitivity to study drug or its excipients;
  • have current signs and symptoms of systemic lupus erythematosus, or severe, progressive, or uncontrolled renal, hepatic, hematologic, endocrine, pulmonary, cardiac, neurologic, or cerebral diseases;
  • have a known history of demyelinating disease suggestive of multiple sclerosis or optic neuritis;
  • presence of a transplanted organ (with the exception of a corneal transplant >3 months prior to Screening);
  • have a history of lymphoproliferative disease including lymphoma, or signs and symptoms suggestive of possible lymphoproliferative disease, such as lymphadenopathy of unusual size or location (eg, nodes in the posterior triangle of the neck, infra-clavicular, epitrochlear, or periaortic areas), or splenomegaly;
  • have any current known malignancy or malignancy within 5 years prior to Screening (except for squamous or basal cell carcinoma of the skin that has been treated with no evidence of recurrence);
  • have poor tolerability of venipuncture or lack of adequate venous access for required blood sampling and infusion of study drug during the study period;
  • have had a known substance abuse or dependency (drug or alcohol) within 3 years of Screening;
  • require chronic (>=1 month) and frequent use (>=3 days per week)of nonsteroidal anti-inflammatory drugs (NSAIDs) except low-dose aspirin for prevention of heart attacks, unstable angina, or transient ischemic attacks;
  • have other inflammatory diseases that might interfere with the evaluation of the ulcerative colitis;
  • have a history of latent or active granulomatous infection, including TB, histoplasmosis, or coccidioidomycosis, prior to Screening.
  • have had a Bacille Calmette-Guerin (BCG) vaccination within 12 months of Screening.
  • have had a chest X-ray within the 3 months prior to the first administration of study agent that shows an abnormality suggestive of malignancy or current active infection, including TB.
  • have received any specified prohibited treatment more recently than the indicated washout period prior to Screening;
  • who are participating in any other clinical study or who have received treatment with any investigational drug or device within 3 months prior Screening;
  • who is part of the staff or a family member of the staff personnel directly involved with this study.

About this study

Official title: Comparison of the Efficacy and Safety of Infliximab, as Monotherapy or in Combination With Azathioprine, Versus Azathioprine Monotherapy in Moderate to Severe Active Ulcerative Colitis (Part 1) Comparison of Maintenance Versus Intermittent Infliximab Treatment in Maintaining Remission: A Follow-Up of Efficacy and Safety (Part 2)

Part 1 of this study is a 3-arm, randomized, active-controlled, parallel-group, multicenter, double-blind, double-dummy, 16-week study to compare the efficacy and safety of infliximab (IFX), as monotherapy or in combination with azathioprine (AZA) versus AZA monotherapy in adults with moderate to severe active ulcerative colitis (UC). Participants who qualify at the Baseline Visit will be eligible to be randomized to one of the three active treatment groups.

Participants in the IFX/AZA combination therapy and IFX monotherapy cohorts will receive IFX infusions at Weeks 0, 2, and 6 and daily oral AZA/placebo, respectively; participants in the AZA cohort will receive daily oral AZA and placebo infusions at Weeks 0, 2, and 6. At Week 8, all participants will be evaluated for response.

Read more

Participants responding to IFX treatment at Week 8, either as monotherapy or in combination with AZA, will receive one more IFX infusion at Week 14; non-responders to IFX therapy will receive placebo infusions at Weeks 8 and 10 and one additional IFX infusion at Week 14.

Participants responding to AZA monotherapy at Week 8 will continue on AZA therapy and receive one placebo infusion at Week 14; nonresponders to AZA will be eligible to receive IFX at Weeks 8, 10, and 14.

Part 2: Participants in remission on IFX monotherapy or IFX/AZA treatment at Week 16 will be randomized to either maintenance or intermittent open-label IFX treatment; randomization will be stratified based on oral AZA/placebo treatment in Part 1. Oral AZA/placebo treatment will continue to be double-blinded. All participants will continue to receive oral AZA/ placebo for the duration of the study.

Participants randomized to maintenance IFX treatment will receive scheduled IFX infusions every 8 weeks beginning at Week 22 (Week 6 for direct entry). If participants lose response, or if treatment has to be discontinued because of an adverse event, these participants are considered treatment failures, and should be followed up for safety at the scheduled 6-month visits (Weeks 38, 62, and 94 [Weeks 22, 46, and 78 for direct entry]). These participants will receive standard of care per their personal physician.

Participants randomized to intermittent IFX treatment will be evaluated every 8 weeks. Participants will receive IFX only upon relapse of disease. Treatment with IFX will be initiated at Weeks 0, 2, and 6 of the individual treatment cycle and will continue every 8 weeks until remission is regained. Throughout the study, individual treatment cycles will be repeated whenever a subject relapses.

In addition, to facilitate enrollment into Part 2, participants who received treatment outside of Part 1 and who are in remission on IFX with or without AZA/6-mercaptopurine (6-MP) will be allowed to enter directly into Part 2. In the Czech Republic, direct entry into Part 2 of the study is not allowed.

A higher than expected incidence of serious infusion reactions observed in the intermittent treatment arm of another study (Protocol P04563, NCT0358670) conducted in participants with moderate to severe psoriasis resulted in the termination of that study. Based on the similarities in study design between the intermittent treatment arm of P04563 and the intermittent treatment arm of Part 2 of this study, enrollment to Part 2 of this study was put on hold, for precautionary reasons. At the same time, all participants already enrolled in the intermittent treatment arm of Part 2 were asked to discontinue from the trial. In October 2009, a decision was made by the sponsor to terminate the whole study (Part 1 and 2). At that time, participants enrolled in Part 1 of the study were allowed to complete their treatment up to Week 16.

Being tested
Infliximab (IFX), Azathioprine (AZA), Placebo to Azathioprine, Placebo infusion
Medical subject headings
Colitis, Ulcerative

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Infliximab (IFX)Biological

    Also called Remicade

    IFX intravenous (IV) infusion dosed at 5mg/kg of body weight

  • Azathioprine (AZA)Drug

    Also called Imuran

    AZA 50 mg tablet dosed at 2.5 mg/kg of body weight orally every day

  • Placebo to AzathioprineDrug

    Placebo to AZA tablet orally every day

  • Placebo infusionDrug

    Placebo to IFX intravenous (IV) infusion

Study groups in NCT00537316
Study groupRoleWhat they receive
Infliximab (IFX)ExperimentalPart 1: IFX 5 mg/kg of body weight intravenous (IV) infusions was to be administered at Weeks 0, 2, and 6 and placebo to AZA was to be taken orally every day for 16 weeks. Responders to IFX at Week 8, were to receive one more IFX infusion at Week 14; non-responders to IFX were to receive placebo IFX infusions at Weeks 8 and 10 and an additional IFX infusion at Week 14. Part 2: Participants in steroid-free remission at Week 16 of Part 1 were to be randomized to either maintenance (every 8 weeks [q8w]) or intermittent (upon relapse) open-label IFX (last IFX infusion administered at Week 86) plus double-blind oral AZA/placebo treatment as allocated in Part 1 (last dose at the final visit, Week 94). Responders at Week 16 who had not achieved steroid-free remission were to continue to receive IFX infusions every 8 weeks.
Receives: Biological: Infliximab (IFX); Drug: Placebo to Azathioprine; Drug: Placebo infusion
Azathioprine (AZA)Active comparatorAZA 2.5 mg/kg of body weight orally every day for 16 weeks. Responders to AZA monotherapy at Week 8 were to continue on AZA therapy and receive one placebo infusion at Week 14; non-responders to AZA at Week 8 would be eligible to receive an IFX infusion at Weeks 8, 10, and 14. Participants in steroid-free remission at Week 16 were to continue on AZA monotherapy and were to be followed up for safety in Part 2. Participants who experienced a relapse of disease after Week 16 were to continue daily AZA monotherapy and receive 3 infusions of IFX (induction therapy at Weeks 0, 2, and 6) followed by infusions every 8 weeks (maintenance therapy).
Receives: Drug: Azathioprine (AZA); Drug: Placebo infusion
IFX/AZAExperimentalIFX 5 mg/kg of body weight IV infusions at Weeks 0, 2, and 6 plus AZA 2.5 mg/kg orally every day for 16 weeks. Responders to IFX/AZA at Week 8 were to receive one more IFX infusion at Week 14; non-responders to IFX/AZA were to receive placebo infusions at Weeks 8 and 10 and one additional IFX infusion at Week 14. Part 2: Participants in steroid-free remission at Week 16 of Part 1 were to be randomized to either maintenance (every 8 weeks [q8w]) or intermittent (upon relapse) open-label IFX (last IFX infusion administered at Week 86) plus double-blind oral AZA/placebo treatment as allocated in Part 1 (last dose at the final visit, Week 94). Responders at Week 16 who had not achieved steroid-free remission were to continue to receive IFX infusions every 8 weeks.
Receives: Biological: Infliximab (IFX); Drug: Azathioprine (AZA); Drug: Placebo infusion
Maintenance IFX/AZA (during Part 2)ExperimentalParticipants randomized to maintenance IFX/AZA in Part 2 of the study were to receive IFX 5 mg/kg of body weight IV infusions every 8 weeks (beginning at Week 22, Week 6 for direct entry) plus AZA 2.5 mg/kg of body weight daily. Four participants were from Part 1 of the study and 1 participant was enrolled directly into Part 2 of the study.
Receives: Biological: Infliximab (IFX); Drug: Azathioprine (AZA)
Maintenance IFX (during Part 2)ExperimentalParticipants randomized to maintenance IFX were to receive IFX 5 mg/kg of body weight IV infusions every 8 weeks (beginning at Week 22, Week 6 for direct entry) in Part 2 of the study. Placebo to AZA therapy was to continue as allocated in Part 1 of the study. All participants were from Part 1 of the study.
Receives: Biological: Infliximab (IFX)
Intermittent IFX/AZA (during Part 2)ExperimentalParticipants randomized to intermittent IFX/AZA were to receive IFX 5 mg/kg of body weight IV infusions only upon relapse of disease (initiated at Weeks 0, 2, and 6 of individual treatment cycle and continued every 8 weeks until remission was regained) plus AZA 2.5 mg/kg of body weight daily in Part 2 of the study. Three participants were from Part 1 of the study and 1 participant was enrolled directly into Part 2 of the study.
Receives: Biological: Infliximab (IFX); Drug: Azathioprine (AZA)
Intermittent IFX (during Part 2)ExperimentalParticipants randomized to intermittent IFX were to receive IFX 5 mg/kg of body weight IV infusions only upon relapse of disease (initiated at Weeks 0, 2, and 6 of individual treatment cycle and continued every 8 weeks until remission was regained). Placebo to AZA therapy was to continue as allocated in Part 1 of the study. One participant was from Part 1 of the study and 1 participant was enrolled directly into Part 2 of the study.
Receives: Biological: Infliximab (IFX)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Proportion of Participants in Steroid-free Remission at Week 16

    Steroid-free remission is defined as a total Mayo score of 2 points or lower, with no individual sub-score exceeding 1 point, without the use of corticosteroids. The total Mayo score consists of the following 4 sub-scores: stool frequency, rectal bleeding, findings of endoscopy (sigmoidoscopy), and physician's global assessment.

    Measured: 16 weeks

  2. Average Remission Rate During Part 2 of the Study

    Due to the early termination of this study, evaluations of efficacy were not conducted for Part 2 of the study (Week 38 through Week 94 [Week 22 through Week 78 for direct entry]).

    Measured: up to Week 94 (Week 78 for direct entry)

Secondary outcomes (4)
  1. Proportion of Participants in Response at Weeks 8 and 16

    Response at Week 8 is defined as a decrease in the partial Mayo score of ≥1 point. Response at Week 16 is defined as a decrease in total Mayo score of ≥3 points and at least 30% lower than baseline Mayo score. The partial Mayo score consists of the following 3 sub-scores: stool frequency, rectal bleeding, and physician's global assessment. The total Mayo score consists of the following 4 sub-scores: stool frequency, rectal bleeding, findings of endoscopy (sigmoidoscopy), and physician's global assessment.

    Measured: Weeks 8 and 16

  2. Proportion of Participants With Mucosal Healing at Week 16

    Mucosal healing was defined as a Mayo endoscopy score of 0 or 1.

    Measured: 16 weeks

  3. Proportion of Participants Who Are in Steroid-free Remission During Part 2 of the Study

    Due to the early termination of this study, evaluations of efficacy were not conducted for Part 2 of the study.

    Measured: Weeks 38, 62 & 94 (Weeks 22, 46 and 78 for direct entry)

  4. Proportion of Participants With Mucosal Healing During Part 2 of the Study

    Due to the early termination of this study, evaluations of efficacy were not conducted for Part 2 of the study

    Measured: Weeks 38, 62 and 94 (Weeks 22, 46 and 78 for direct entry)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Oct 2007 (estimated)
  2. Study startJul 2007
  3. Primary completionFeb 2010
  4. Study completionFeb 2010

Why it stopped: Infusion reactions during re-induction cycles after a period of no treatment in another study \[P04563, NCT0358670\]

Resources

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
Double blind
Who is masked
Participant, Investigator
Enrolment
242 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00537316
Sponsor's protocol ID
P04807
Status last verified
Mar 2017
First submitted
28 Sep 2007
Last updated
12 Apr 2017
Data monitoring committee
No
Individual data sharing
Yes: http://www.merck.com/clinical-trials/pdf/Merck%20Procedure%20on%20Clinical%20Trial%20Data%20Access%20Final\_Updated%20July\_9\_2014.pdf http://engagezone.msd.com/ds\_documentation.php

Publications

  • Panaccione R, Ghosh S, Middleton S, Marquez JR, Scott BB, Flint L, van Hoogstraten HJ, Chen AC, Zheng H, Danese S, Rutgeerts P. Combination therapy with infliximab and azathioprine is superior to monotherapy with either agent in ulcerative colitis. Gastroenterology. 2014 Feb;146(2):392-400.e3. doi: 10.1053/j.gastro.2013.10.052.

    PubMed 24512909
  • Hasskamp J, Meinhardt C, Patton PH, Timmer A. Azathioprine and 6-mercaptopurine for maintenance of remission in ulcerative colitis. Cochrane Database Syst Rev. 2025 Feb 27;2(2):CD000478. doi: 10.1002/14651858.CD000478.pub5.

    PubMed 40013523

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Infliximab (IFX) and Azathioprine (AZA) in people with Ulcerative Colitis, run by Merck Sharp & Dohme LLC.

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 21 inclusion and 37 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: stopped early, as of the sponsor's last update on 12 Apr 2017. 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?

Yes. Results are posted in the registry (first posted 16 Dec 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Proportion of Participants in Steroid-free Remission at Week 16 (time frame: 16 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00537316, which also lists 2 linked publications.

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 12 Apr 2017.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in