Clinical trial NCT05470985: A Study to Evaluate the Efficacy, Safety, and Drug Levels of Oral Ozanimod in Pediatric Participants With Moderately to Severely Active Crohn's Disease With an Inadequate Response to Conventional Therapy
Official title: A Phase 2/3, Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Oral Ozanimod (RPC1063) in Pediatric Participants With Moderately to Severely Active Crohn's Disease With an Inadequate Response to Conventional Therapy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Crohn Disease
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2022-07-22
Enrollment: 5
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy, safety, drug levels, and drug effects of ozanimod in pediatric participants with moderately to severely active Crohn's Disease.


Eligibility criteria: Inclusion Criteria:

* Participants must have a Pediatric Crohn's Disease Activity Index (PCDAI) score ≥ 30 and a Simple Endoscopic Score for Crohn's Disease (SES-CD) score ≥ 6 (or SES-CD ≥ 4 in participants with isolated ileal disease)
* Participant has an inadequate response, intolerance, or loss of response to at least 1 of the following treatments for Crohn's Disease (CD):

  i) corticosteroids ii) immunomodulators iii) biologic therapy iv) other systemic immunomodulatory therapies for CD

Exclusion Criteria:

* Participant is likely to require, in the physician's judgment, bowel resection within 12 weeks of entry into the study
* Participant has current stoma, ileal-anal pouch anastomosis, fistula that is likely to require, in the physician's judgment, surgical or medical intervention within 12 weeks of entry into the study or need for ileostomy or colostomy
* Participant has extensive small bowel resection (\> 100 cm) or known diagnosis of short bowel syndrome or participant requires total parenteral nutrition

Other protocol-defined inclusion/exclusion criteria apply.

Primary outcomes:
measure: Percentage of Participants Who Achieve Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64
timeFrame: Week 64
measure: Percentage of Participants Achieving Simple Endoscopic Score for Crohn's Disease (SES-CD) ≤ 2 or SES-CD ≤ 4 Points With no SES-CD Subscore > 1 Point at Week 64
timeFrame: Week 64
Secondary outcomes:
measure: Percentage of Participants Who Achieve Pediatric Crohn's Disease Activity Index (PCDAI) < 10 at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Percentage of Participants Who Achieve Simple Endoscopic Score for Crohn's Disease (SES-CD) Decrease From Baseline of ≥ 50% (ER-50) at Week 64
timeFrame: Week 64
measure: Percentage of Participants Who Achieve Simple Endoscopic Score for Crohn's Disease (SES-CD) Decrease From Baseline of ≥ 50% (ER-50) at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Percentage of Participants Who Achieve Reduction in Pediatric Crohn's Disease Activity Index (PCDAI) Score ≥ 12.5 and a Total PCDAI Score of < 30 Points at Week 64
timeFrame: Week 64
measure: Percentage of Participants Who Achieve Reduction in Pediatric Crohn's Disease Activity Index (PCDAI) Score ≥ 12.5 and a Total PCDAI Score of < 30 Points at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Percentage of Adolescents Who Achieve an Average Daily Abdominal Pain Score ≤ 1 Point and an Average Daily Stool Frequency ≤ 3 Points With Abdominal Pain and Stool Frequency no Worse Than Baseline at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Percentage of Adolescents Who Achieve an Average Daily Abdominal Pain Score ≤ 1 Point and an Average Daily Stool Frequency ≤ 3 Points With Abdominal Pain and Stool Frequency no Worse Than Baseline at Week 64
timeFrame: Week 64
measure: Change From Baseline in Stool Frequency (SF) Score at Week 64
timeFrame: Baseline and Week 64
measure: Change From Baseline in Abdominal Pain (AP) Score at Week 64
timeFrame: Baseline and Week 64
measure: Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Score < 150 at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Score < 150 at Week 64
timeFrame: Week 64
measure: Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Reduction From Baseline of ≥ 100 Points or CDAI Score < 150 at Week 64
timeFrame: Week 64
measure: Percentage of Adolescents Who Achieve Crohn's Disease Activity Index (CDAI) Reduction From Baseline of ≥ 100 Points or CDAI Score < 150 at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Percentage of Participants Who Achieve a Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64 While Remaining Corticosteroid Free in the Prior 12 Weeks
timeFrame: Week 64
measure: Percentage of Adolescents Who Achieve a Pediatric Crohn's Disease Activity Index (PCDAI) Score < 10 at Week 64 While Remaining Corticosteroid Free in the Prior 12 Weeks
timeFrame: Week 64
measure: Percentage of Participants Who Achieve a Crohn's Disease Activity Index (CDAI) Score < 150 at Week 64 While Remaining Corticosteroid Free in the Prior 12 Weeks
timeFrame: Week 64
measure: Percentage of Participants Achieving Simple Endoscopic Score for Crohn's Disease (SES-CD) ≤ 2 or SES-CD ≤ 4 Points With no SES-CD Subscore > 1 Point at Week 12 Based on Data as Observed
timeFrame: Week 12
measure: Steady State Systemic Exposures of Ozanimod at Week 20 and Throughout the Study
timeFrame: Week 20 and up to end of study (Week 64)
measure: Absolute Lymphocyte Count at Week 12 Based on Data as Observed
timeFrame: Baseline (Day 1) and Week 12
measure: Absolute Lymphocyte Count at Week 64
timeFrame: Week 64
measure: Number of Participants With Adverse Events
timeFrame: From first dose (Day 1) and up to Week 12
Other outcomes:
Locations:
city: Garden Grove
country: United States
facility: Local Institution - 0010
state: California
city: Los Angeles
country: United States
facility: Local Institution - 0028
state: California
city: Sacramento
country: United States
facility: Local Institution - 0002
state: California
city: San Francisco
country: United States
facility: Local Institution - 0009
state: California
city: Hartford
country: United States
facility: Local Institution - 0011
state: Connecticut
city: Orlando
country: United States
facility: Local Institution - 0044
state: Florida
city: Orlando
country: United States
facility: Local Institution - 0037
state: Florida
city: Atlanta
country: United States
facility: Local Institution - 0053
state: Georgia
city: Indianapolis
country: United States
facility: Local Institution - 0059
state: Indiana
city: Springfield
country: United States
facility: Local Institution - 0038
state: Massachusetts
city: Detroit
country: United States
facility: Local Institution - 0006
state: Michigan
city: Rochester
country: United States
facility: Local Institution - 0003
state: Minnesota
city: Saint Louis
country: United States
facility: Local Institution - 0016
state: Missouri
city: Lebanon
country: United States
facility: Local Institution - 0071
state: New Hampshire
city: New York
country: United States
facility: Local Institution - 0060
state: New York
city: Fort Worth
country: United States
facility: Local Institution - 0075
state: Texas
city: Houston
country: United States
facility: Local Institution - 0070
state: Texas
city: Seattle
country: United States
facility: Local Institution - 0008
state: Washington
city: Tacoma
country: United States
facility: Local Institution - 0040
state: Washington
city: Joonladup
country: Australia
facility: Local Institution - 0045
state: Western Australia
city: Brussel
country: Belgium
facility: Local Institution - 0058
state: BRU
city: Leuven
country: Belgium
facility: Local Institution - 0023
state: VBR
city: Liege
country: Belgium
facility: Local Institution - 0062
state: WLG
city: Liège
country: Belgium
facility: Local Institution - 0048
state: WLG
city: Brussel
country: Belgium
facility: Local Institution - 0047
city: Bruxelles
country: Belgium
facility: Local Institution - 0055
city: Halifax
country: Canada
facility: Local Institution - 0014
state: Nova Scotia
city: London
country: Canada
facility: Local Institution - 0001
state: Ontario
city: Montreal
country: Canada
facility: Local Institution - 0054
state: Quebec
city: Bron
country: France
facility: Local Institution - 0066
city: Caen
country: France
facility: Local Institution - 0065
city: Paris
country: France
facility: Local Institution - 0072
city: Toulouse
country: France
facility: Local Institution - 0063
city: Dresden
country: Germany
facility: Local Institution - 0057
state: SN
city: Leipzig
country: Germany
facility: Local Institution - 0067
state: SN
city: Miskolc
country: Hungary
facility: Local Institution - 0015
city: Szeged
country: Hungary
facility: Local Institution - 0026
city: Warszawa
country: Poland
facility: Local Institution - 0007
state: MZ
city: Rzeszow
country: Poland
facility: Local Institution - 0052
state: PK
city: Lodz
country: Poland
facility: Local Institution - 0061
city: Warsaw
country: Poland
facility: Local Institution - 0033
city: Warszawa
country: Poland
facility: Local Institution - 0031
city: Barcelona
country: Spain
facility: Local Institution - 0025
state: B
city: Badalona
country: Spain
facility: Local Institution - 0030
city: Barcelone
country: Spain
facility: Local Institution - 0035
city: Madrid
country: Spain
facility: Local Institution - 0017
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: