Clinical trial • Phase II/III • Gastroenterology

OZANIMOD for Ulcerative colitis

Phase II/III trial of OZANIMOD for Ulcerative colitis. Randomised. 66 participants.

Overview

Trial Therapeutic Area
Gastroenterology
Trial Disease
Ulcerative colitis
Trial Stage
Phase II/III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
19-02-2024
First CTIS Authorization Date
15-03-2024

Trial design

Randomised Phase II/III trial across 20 sites in Spain, France, Belgium and others.

Randomised
Yes
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
66
Trial Duration For Participant
364

Eligibility

Recruits 66 paediatric patients.

Vulnerable Population
Pediatric subjects are included (isVulnerablePopulationSelected = true). Assent and parental/guardian consent processes are provided: pediatric assent forms for age groups (e.g., Assent Age 12-15, Assent Age 16-17) and parent/guardian consent forms and information sheets are available. Multiple language versions of ICF/assent documents are present (examples: English, Spanish, French, German, Polish, Dutch).

Inclusion criteria

  • {"criterion_text":"- Moderately to severely active Ulcerative Colitis (UC) diagnosed prior to the Screening Visit\n- Evidence of UC extending beyond the rectum, as determined by baseline endoscopy\n- Has had an inadequate response, loss of response to, or is intolerant to at least 1 of the following treatments for UC: oral aminosalicylates, systemic corticosteroids, immunomodulators, biologic therapy"}

Exclusion criteria

  • {"criterion_text":"- Diagnosis of Crohn's disease or indeterminate colitis\n- Has documentation of positive test for toxin producing Clostridium difficile, or polymerase chain reaction examination of the stool\n- Apheresis within 2 weeks of randomization\n- History of or currently active primary or secondary immunodeficiency, or participants with known genetic disorders as a cause for colitis"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of subjects who achieve clinical remission, defined as all the following: • Mayo Endoscopy Subscore (MES) ≤ 1 • Rectal Bleeding Subscore (RBS) = 0 • Stool Frequency Subscore (SFS) ≤ 1, with a decrease of ≥ 1 point from Baseline SFS","definition_or_measurement_approach":"Clinical remission defined by composite 3-component Mayo score elements: MES ≤ 1; RBS = 0; SFS ≤ 1 with ≥1 point decrease from Baseline SFS (measured by endoscopy and symptom subscores)."}
  • {"endpoint_text":"- Proportion of subjects who achieve histological remission, defined as the following: - Geboes index score < 2.0 - Robarts Histopathology Index (RHI) ≤ 3, with subscores of 0 for both lamina propria neutrophils and neutrophils in the epithelium","definition_or_measurement_approach":"Histological remission defined by Geboes index <2.0 and RHI ≤3 with subscores of 0 for lamina propria neutrophils and neutrophils in the epithelium (histopathology assessment)."}
  • {"endpoint_text":"- Early Discontinuation: Starting at Week 5 or later if either one of the following criteria are met at 2 consecutive visits that are at least 2 weeks apart: - Partial Mayo score is ≥ 7 and is the same or worse than baseline, AND rectal bleeding and stool frequency are not improved from baseline - Partial Mayo score is 6 and worse than baseline, AND rectal bleeding and stool frequency are not improved from baseline","definition_or_measurement_approach":"Early discontinuation defined by partial Mayo score thresholds assessed at two consecutive visits ≥2 weeks apart (criteria as stated)."}

Secondary endpoints

  • {"endpoint_text":"- Proportion of subjects who achieve clinical response defined as all the following: • Decrease from Baseline in the 3-component Mayo score of at least 2 points and at least 35% • Decrease in RBS of at least 1 point OR absolute RBS ≤ 1","definition_or_measurement_approach":"Clinical response defined by decrease from Baseline in 3-component Mayo score (≥2 points and ≥35%) and specified change in RBS (≥1 point decrease or absolute RBS ≤1)."}
  • {"endpoint_text":"- Proportion of subjects who achieve clinical remission defined as all the following: • MES ≤ 1 • RBS = 0 • SFS ≤ 1, with a decrease of ≥1 point from Baseline SFS","definition_or_measurement_approach":"Same clinical remission definition as primary but listed as a secondary endpoint for different timepoints (MES, RBS, SFS criteria measured by endoscopy and symptom subscores)."}
  • {"endpoint_text":"- Proportion of subjects who achieve symptomatic improvement of UC, defined as all the following: • RBS = 0 • SFS ≤ 1 • Decrease in SFS of ≥ 1 from Baseline","definition_or_measurement_approach":"Symptomatic improvement defined by RBS=0, SFS≤1 and ≥1 point decrease in SFS from Baseline (patient-reported symptoms)."}
  • {"endpoint_text":"- Proportion of subjects who achieve endoscopic improvement, defined as MES ≤ 1","definition_or_measurement_approach":"Endoscopic improvement defined by Mayo Endoscopy Subscore (MES) ≤1 assessed by endoscopy."}
  • {"endpoint_text":"- Proportion of subjects who achieve corticosteroid-free remission at Week 52, defined as all the following: • Did not receive steroids for ≥ 12 weeks prior to Week 52 • Achieved clinical remission defined by the 3-component Mayo score","definition_or_measurement_approach":"Corticosteroid-free remission defined by no steroid use for ≥12 weeks prior to Week 52 and achievement of clinical remission per 3-component Mayo score."}
  • {"endpoint_text":"- Secondary – Safety and Tolerability Number and proportion of subjects experiencing AEs, SAEs, AEs leading to discontinuation from treatment, and AEs of interest (AEIs)","definition_or_measurement_approach":"Safety/tolerability measured by counts and proportions of adverse events (AEs), serious AEs (SAEs), AEs leading to discontinuation, and AEs of interest (AESIs)."}
  • {"endpoint_text":"- Secondary - Pharmacokinetics Steady state systemic exposure of ozanimod and CC112273","definition_or_measurement_approach":"Pharmacokinetics measured as steady-state systemic exposure (e.g., PK parameters) of ozanimod and metabolite CC112273."}
  • {"endpoint_text":"- Secondary – Pharmacodynamics Absolute and percent change from baseline in ALC","definition_or_measurement_approach":"Pharmacodynamic assessment via absolute and percent change from baseline in absolute lymphocyte count (ALC)."}

Recruitment

Planned Sample Size
66
Recruitment Window Months
142
Consent Approach
Informed consent is managed with parent/guardian consent and pediatric assent. Age-specific assent documents are provided (examples: Assent Age 12-15, Assent Age 16-17) alongside parent/guardian information and consent forms. Multiple language versions of ICF and assent documents are available (documentation present in English, Spanish, French, German, Polish, Dutch and country-specific versions).

Geography

Total Number Of Sites
20
Total Number Of Participants
62

Spain

Earliest CTIS Part Ii Submission Date
01-03-2024
Latest Decision Or Authorization Date
25-03-2025
Processing Time Days
389
Number Of Sites
4
Number Of Participants
10

Sites

Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
Servicio de Gastroenterologia
Principal Investigator Name
Javier Martin de Carpi
Principal Investigator Email
javier.martinc@sjd.es
Contact Person Name
Javier Martin de Carpi
Contact Person Email
javier.martinc@sjd.es
Site Name
Hospital Infantil Universitario Nino Jesus
Department Name
Gastroenterology
Principal Investigator Name
Laura Palomino
Principal Investigator Email
laura_palomino_perez@hotmail.es
Contact Person Name
Laura Palomino
Site Name
Hospital Germans Trias I Pujol
Department Name
Unidad de Gastroenterologia
Principal Investigator Name
Montserrat Montraveta
Principal Investigator Email
mmontraveta.germanstrias@gencat.cat
Contact Person Name
Montserrat Montraveta
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Pediatric Gastroenterology, Hepatology and Nutrition Unit
Principal Investigator Name
Cesar Sanchez
Principal Investigator Email
sangoros@hotmail.com
Contact Person Name
Cesar Sanchez
Contact Person Email
sangoros@hotmail.com

France

Earliest CTIS Part Ii Submission Date
01-03-2024
Latest Decision Or Authorization Date
04-12-2025
Processing Time Days
644
Number Of Sites
4
Number Of Participants
16

Sites

Site Name
Centre Hospitalier Universitaire De Caen Normandie
Department Name
Pediatric Gastroenterology
Principal Investigator Name
Claire DUPONT-LUCAS
Principal Investigator Email
dupont-c@chu-caen.fr
Contact Person Name
Claire DUPONT-LUCAS
Contact Person Email
dupont-c@chu-caen.fr
Site Name
Hospital Femme Mere Enfant
Department Name
Hepato-Gastroenterology and Pediatric Nutrition
Principal Investigator Name
Rémi DUCLAUX-LORAS
Principal Investigator Email
remi.duclaux-loras@chu-lyon.fr
Contact Person Name
Rémi DUCLAUX-LORAS
Contact Person Email
remi.duclaux-loras@chu-lyon.fr
Site Name
Hopital Necker Enfants Malades
Department Name
Pediatric Gastroenterology and Nutrition
Principal Investigator Name
Franck RUEMMELE
Principal Investigator Email
frank.ruemmele@aphp.fr
Contact Person Name
Franck RUEMMELE
Contact Person Email
frank.ruemmele@aphp.fr
Site Name
Hopital Des Enfants
Department Name
Gastroenterology, Hepatology, Nutrition and Diseases Hereditary Metabolism
Principal Investigator Name
Emmanuel MAS
Principal Investigator Email
mas.e@chu-toulouse.fr
Contact Person Name
Emmanuel MAS
Contact Person Email
mas.e@chu-toulouse.fr

Belgium

Earliest CTIS Part Ii Submission Date
01-03-2024
Latest Decision Or Authorization Date
03-12-2025
Processing Time Days
643
Number Of Sites
4
Number Of Participants
8

Sites

Site Name
UZ Leuven
Department Name
Pediatric gastroenterology
Principal Investigator Name
Ilse Hoffman
Principal Investigator Email
ilse.hoffman@uzleuven.be
Contact Person Name
Ilse Hoffman
Contact Person Email
ilse.hoffman@uzleuven.be
Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Pediatric gastroenterology
Principal Investigator Name
Emeline Bequet
Principal Investigator Email
emeline.bequet@citadelle.be
Contact Person Name
Emeline Bequet
Contact Person Email
emeline.bequet@citadelle.be
Site Name
UZ Brussel
Department Name
Pediatric gastroenterology
Principal Investigator Name
Elisabeth De Greef
Principal Investigator Email
elisabeth.degreef@uzbrussel.be
Contact Person Name
Elisabeth De Greef
Contact Person Email
elisabeth.degreef@uzbrussel.be
Site Name
Universitair Ziekenhuis Antwerpen
Department Name
Dept of Paediatric Gastroenterology
Principal Investigator Name
Nicolette Moes
Principal Investigator Email
nicolette.moes@uza.be
Contact Person Name
Nicolette Moes
Contact Person Email
nicolette.moes@uza.be

Poland

Earliest CTIS Part Ii Submission Date
01-03-2024
Latest Decision Or Authorization Date
08-12-2025
Processing Time Days
648
Number Of Sites
5
Number Of Participants
20

Sites

Site Name
Instytut Pomnik Centrum Zdrowia Dziecka
Department Name
Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii
Principal Investigator Name
Jarosław Kierkuś
Principal Investigator Email
j.kierkus@med-net.pl
Contact Person Name
Jarosław Kierkuś
Contact Person Email
j.kierkus@med-net.pl
Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Department Name
Klinika Pediatrii, Gastroenterologii i Żywienia
Principal Investigator Name
Tomasz Pytrus
Principal Investigator Email
tomasz.pytrus@umw.edu.pl
Contact Person Name
Tomasz Pytrus
Contact Person Email
tomasz.pytrus@umw.edu.pl
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Department Name
Uniw. Centrum Pediatrii Klinika Alergologii Gastroenterologii i Żywienia Dzieci ul. Pankiewicza 16
Principal Investigator Name
Ewa Toporowska-Kowalska
Principal Investigator Email
ewa.toporowska-kowalska@umed.lodz.pl
Contact Person Name
Ewa Toporowska-Kowalska
Site Name
Twoja Przychodnia Szczecinskie Centrum Medyczne Sp. z o.o.
Principal Investigator Name
Beata Gawdis-Wojnarska
Principal Investigator Email
gawdis@twojaprzychodnia.com
Contact Person Name
Beata Gawdis-Wojnarska
Contact Person Email
gawdis@twojaprzychodnia.com
Site Name
Medical Network Sp. z o.o.
Department Name
WIP Warsaw IBD Point Profesor Kierkuś
Principal Investigator Name
Monika Meglicka
Principal Investigator Email
m.meglicka@wip.waw.pl
Contact Person Name
Monika Meglicka
Contact Person Email
m.meglicka@wip.waw.pl

Germany

Earliest CTIS Part Ii Submission Date
01-03-2024
Latest Decision Or Authorization Date
04-12-2025
Processing Time Days
644
Number Of Sites
3
Number Of Participants
8

Sites

Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Kinderklinik des Dr. v. Haunerschen Kinderspitals
Principal Investigator Name
Tobias Schwerd
Principal Investigator Email
tobias.schwerd@med.uni-muenchen.de
Contact Person Name
Tobias Schwerd
Site Name
Universitaet Leipzig
Department Name
Klinik und Poliklinik fuer Kinder- und Jugendmedizin
Principal Investigator Name
Gunter Flemming
Principal Investigator Email
gunter.flemming@medizin.uni-leipzig.de
Contact Person Name
Gunter Flemming
Site Name
Technische Universitat Dresden
Department Name
CMCB, Center f. Regen. Therapies (AG Bonifacio)
Principal Investigator Name
Martin Laaß
Principal Investigator Email
martin.laass@uniklinikum-dresden.de
Contact Person Name
Martin Laaß

Sponsor

Primary sponsor

Full Name
Bristol-Myers Squibb Services Unlimited Company
Organisation Type
Pharmaceutical company
Country Of Registered Address
Ireland

Contract research organisations

Name
Iqvia Holdings Inc.
Responsibilities
Monitoring, Regulatory, Investigator recruitment, Data management, SUSAR reporting, Quality assurance auditing, Statistical analysis, Medical writing
Name
Icon Development Solutions LLC
Responsibilities
PK analysis
Name
Signant Health LLC
Responsibilities
eCOA, IVRS – treatment randomisation
Name
PPD Global Clinical Labs
Responsibilities
Sample management, kit building, storage and distribution of samples; biomarker analysis
Name
Biotel Research LLC
Responsibilities
ECG central reading, cardiac and imaging

Third parties

  • {"country":"United States","full_name":"Signant Health LLC","duties_or_roles":"eCOA, IVRS – treatment randomisation","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"PPD Global Clinical Labs","duties_or_roles":"Sample mgmt, Kit building, Storage and distribution of samples to other vendors for analysis. Biomarker analysis will be also be performed","organisation_type":"Industry"}
  • {"country":"United Kingdom","full_name":"Q2 Solutions","duties_or_roles":"biomarker analysis","organisation_type":"Health care"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Provides electronic payments, travel arrangements,electronic study-related comunications to patients","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"Data entry of Local Laboratory Values","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Sample storage","organisation_type":"Pharmaceutical company"}
  • {"country":"Canada","full_name":"Alimentiv Inc.","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, etc","organisation_type":"Pharmaceutical company"}
  • {"country":"India","full_name":"Accenture Solutions Private Limited","duties_or_roles":"Embarc operations","organisation_type":"Industry"}
  • {"country":"United States","full_name":"Iqvia Holdings Inc.","duties_or_roles":"Monitoring Regulatory Investigator recruitment Data management SUSAR reporting Quality assurance auditing Statistical analysis Medical writing","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"PK analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"Denmark","full_name":"Nordic Bioscience A/S","duties_or_roles":"Speciality Biomarker Laboratory","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Biotel Research LLC","duties_or_roles":"ECG Central reading, cardiac and imaging","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Ozanimod
Active Substance
OZANIMOD
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL
Authorisation Status
Authorised

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