Safety and Efficacy Trial of RPC1063 for Moderate to Severe Ulcerative Colitis

• Register: ClinicalTrials.gov
• Last refreshed on: 8 March 2021
• Main ID: NCT02435992
• Date of registration: 24/04/2015
• Prospective Registration: Yes
• Primary sponsor: Celgene
• Public title: Safety and Efficacy Trial of RPC1063 for Moderate to Severe Ulcerative Colitis
• Scientific title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Oral RPC1063 as Induction and Maintenance Therapy for Moderate to Severe Ulcerative Colitis
• Date of first enrolment: June 17, 2015
• Target sample size: 1012
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02435992
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belarus, Belgium, Bulgaria, Canada, Croatia, Czech Republic, Czechia, Georgia, Germany, Greece, Hungary, Israel, Italy, Korea, Republic of, Latvia, Moldova, Republic of, Netherlands, New Zealand, Poland, Romania, Russian Federation, Serbia, Slovakia, South Africa, Spain, Ukraine, United Kingdom, United States

Contacts

• Name: AnnKatrin Petersen, M.D., MSc.
• Affiliation: Celgene

Key inclusion & exclusion criteria

Inclusion Criteria:

- Aged 18 to 75 years (at screening for Cohort 1 and 2)

- Male or female adolescent patients aged 12 to < 18 years (at screening) with a body
weight greater than or equal to 45 kg

- UC confirmed on endoscopy

- Moderately to severely active UC (May score 6-12)

- Currently receiving treatment with aminosalisylate, prednisone, or budesonide

- Can be receiving azathioprine, mercaptopurine, or methotrexate, but treatment will be
stopped prior to randomization

Exclusion Criteria:

- Have severe extensive colitis as evidence by:

- Physician judgment that the patient is likely to require colectomy or ileostomy within
12 weeks of baseline.

- Current or recent (within 3 months) evidence of fulminant colitis, toxic megacolon, or
bowel perforation.

- Diagnosis of CD, indeterminate colitis, or the presence of fistula consistent with CD
or microscopic colitis, radiation colitis, or ischemic colitis

- Clinically relevant cardiovascular conditions or other relevant diseases that could
impact the implementation or interpretation of the trial, or put the patient at risk

- History of uveitis or unknown macular edema

- Pregnancy, lactation, or a positive serum ß-human chorionic gonadotropin (ß-hCG)
measured during screening



Age minimum: 12 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Ulcerative Colitis

Intervention(s)

Drug: RPC1063

Drug: Placebo

Primary Outcome(s)

The proportion of patients in clinical remission at 52 weeks (Maintenance Period) [Time Frame: 52 weeks]

The proportion of patients in clinical remission at Week 10 (Induction Period) [Time Frame: 10 weeks]

Secondary Outcome(s)

Assessment of Safety and Tolerability [Time Frame: 52 weeks]

The proportion of adult patients with mucosal healing at 52 weeks [Time Frame: 52 weeks]

The proportion of patients with clinical remission at 52 weeks who previously received anti-TNF therapy [Time Frame: 52 weeks]

Change in Complete Mayo Score from Baseline to Week 10 [Time Frame: 10 weeks]

Change in Fecal calprotectin from baseline to 52 weeks [Time Frame: 52 Weeks]

Change in Plasma protein (cytokines, chemokines, and other markers of inflammation) from baseline to 52 weeks [Time Frame: 52 weeks]

Proportion of patients with histologic remission at Week 10 [Time Frame: 10 weeks]

The proportion of adult patients with corticosteroid-free remission [Time Frame: 52 weeks]

The proportion of patients with a clinical response (3-component Mayo with different definitions) at Week 52 [Time Frame: 52 weeks]

The proportion of patients with clinical response at Week 10 who previously received anti-TNF [Time Frame: 10 weeks]

Work productivity at 28 weeks, 40 weeks, and at 52 weeks [Time Frame: 52 weeks]

Change in Complete Mayo score from Baseline to 52 weeks [Time Frame: 52 weeks]

Proportion of patients in clinical remission at Week 10 [Time Frame: 10 weeks]

Absolute lymphocyte count (ALC) derived from blinded hematology laboratory results [Time Frame: 52 weeks]

Change in CRP from baseline to 52 weeks [Time Frame: 52 weeks]

The proportion of adult patients with endoscopic improvement at 52 weeks who previously received anti-TNF therapy [Time Frame: 52 weeks]

Change in Partial Mayo score from Baseline to 52 weeks [Time Frame: 52 weeks]

Change in Partial Mayo score from Baseline to Week 10 [Time Frame: 10 weeks]

Change in 9-point Mayo score from Baseline to Week 10 [Time Frame: 10 weeks]

Health resource utilization at 28 weeks, 40 weeks, and at 52 weeks [Time Frame: 52 weeks]

The proportion of patients with a clinical response at 52 weeks [Time Frame: 52 weeks]

The proportion of patients with a clinical response at Week 10 [Time Frame: 10 weeks]

Change in EQ-5D from Baseline to 52 weeks [Time Frame: 52 weeks]

Change in the SF-36 from Baseline to 52 weeks [Time Frame: 52 weeks]

Work productivity at Week 10 [Time Frame: 10 weeks]

Change in 3-component Mayo score from Baseline to 52 weeks [Time Frame: 52 weeks]

Change in the EQ-5D from Baseline to Week 10 [Time Frame: 10 weeks]

The proportion of patients with durable clinical remission [Time Frame: 52 weeks]

The proportion of patients with histologic remission at 52 weeks [Time Frame: 52 weeks]

Change in the SF-36 from Baseline to Week 10 [Time Frame: 10 weeks]

The proportion of patients with endoscopic improvement at 52 weeks [Time Frame: 52 weeks]

The proportion of adult patients with clinical remission at Week 10 who previously received anti-TNF [Time Frame: 10 weeks]

Health resource utilization at Week 10 [Time Frame: 10 weeks]

Pharmacokinetic (PK) Assessment [Time Frame: 52 weeks]

The proportion of patients with endoscopic improvement at Week 10 [Time Frame: 10 weeks]

The proportion of adult patients in remission at 52 weeks while off corticosteroids for any length of time [Time Frame: 52 weeks]

The proportion of patients in clinical remission at 52 weeks in the subset of patients who were in remission at Week 10 [Time Frame: 52 weeks]

The proportion of patients with clinical response at 52 weeks who previously received anti-TNF therapy [Time Frame: 52 weeks]

The proportion of patients with endoscopic improvement at week 10 who previously received anti-TNF [Time Frame: 10 weeks]

The proportion of patients with mucosal healing at Week 10 [Time Frame: 10 weeks]

The proportion of patients in clinical remission (Four-component Mayo, with different definitions) at Week 52 [Time Frame: 52 weeks]

Secondary ID(s)

RPC01-3101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors