Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Oral Ozanimod in Pediatric Participants with Moderately to Severely Active Crohn’s Disease with an Inadequate Response to Conventional Therapy

Phase 1

• Conditions: Moderately to Severely Active Crohn’s Disease; MedDRA version: 20.0Level: PTClassification code 10011401Term: Crohn's diseaseSystem Organ Class: 10017947 - Gastrointestinal disorders; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2021-005019-30-PL
• Lead Sponsor: Celgene International II Sarl

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-11-04
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
1) Signed Written Informed Consent
2)Type of Participant and Target Disease Characteristics
a) Participant is willing and able to adhere to the study visit schedule and other protocol requirements including is willing and able to swallow a capsule until a sprinkle formulation of ozanimod is available.
b) Participant has been diagnosed with CD = 3 months prior to the Screening Visit. The diagnosis should be confirmed by clinical and endoscopic evidence and corroborated by a histopathology report (Note: Histopathology may be performed during endoscopy at Screening if no prior report is readily available).
c) Participant has met each of the following 2 criteria:
i) A PCDAI score = 30.
ii) Participant has a SES-CD score = 6 (or SES-CD = 4 in participants with isolated ileal disease).
d) Participant has an inadequate response, intolerance, or loss of response to at least 1 of the following treatments for CD.
i) corticosteroids (eg, oral prednisone, budesonide MMX, IV corticosteroids).
ii) immunomodulators (eg, AZA, 6-MP, cyclosporine, MTX).
iii) biologic therapy (eg, ustekinumab, abatacept, infliximab, etanercept, adalimumab, anakinra, rituximab, vedolizumab).
iv) other systemic immunomodulatory therapies for CD.
e) If the participant is taking the following background therapies for CD, a stable dose must be maintained as indicated below (dosage regimen can be adjusted to accommodate mg/kg/day dosing as appropriate):
i) Oral aminosalicylates (eg, mesalamine, sulfasalazine, olsalazine, balsalazide), the dose must have been stable starting 3 weeks prior to Screening endoscopy.
ii) Prednisone (= 0.5 mg/kg/day up to 20 mg/day), the dose must have been stable starting 2 weeks prior to Screening endoscopy and must remain stable through the first 5 weeks of treatment.
iii) Budesonide therapy (doses = 9 mg per day) or beclomethasone (doses = 5 mg per day), the dose must have been stable starting 3 weeks prior to Screening endoscopy and must remain stable through the first 5 weeks of treatment
f) Participant must have documentation of vaccinations per standard immunization schedule including complete varicella vaccination at least 30 days prior to randomization
3) Age of Participant
a) Participant must be age 2 to 17 years, inclusive (for the assigned cohort) at the time of informed consent/assent.

Please refer to Protocol for detailed list of Inclusion criteria
Are the trial subjects under 18? yes
Number of subjects for this age range: 120
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:
1) Medical Conditions
a) Participant has clinically relevant cardiovascular, hepatic, neurological, pulmonary, ophthalmological, endocrine, psychiatric, or other major systemic disease making implementation of the protocol or interpretation of the study difficult or that would put the participant at risk by participating in the study.
i) Clinically relevant pulmonary conditions include, but are not limited to, history of severe or chronic lung disease, bronchopulmonary dysplasia, cystic fibrosis, or severe asthma (ie, that interferes with normal activities of daily living).
b) Participant is likely to require, in the physician’s judgment, bowel resection within 12 weeks of entry into the study.
c) Participant has a diagnosis of UC, indeterminate colitis, radiation colitis, or ischemic colitis, or has strictures with prestenotic dilatation, requiring procedural intervention, or with obstructive symptoms. In addition, participants with colonic or ileal strictures that are not passable with an age-appropriate colonoscope that the endoscopist normally uses in clinical practice, or strictures in the ileum or ileocecal valve that are fibrotic in nature, will be excluded.
d) 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.
e) Participant has extensive small bowel resection (> 100 cm) or known diagnosis of short bowel syndrome or participant requires total parenteral nutrition.
f) Participant has suspected or diagnosed intra-abdominal or perianal abscess that has not been appropriately treated.
g) Participant has documentation of positive test for toxigenic Clostridioides difficile (formerly Clostridium difficile [C difficile]) by polymerase chain reaction examination of the stool during Screening. If positive, participants may be rescreened after appropriate treatment and negative retest no earlier than 7 days after completion of treatment.
h) Participant has documentation of positive examination for pathogens (ova, parasites, and bacteria). If positive, participants may be treated and rescreened.
i) Participant requires or is expected to undergo apheresis (eg, Adacolumn apheresis) within 2 weeks of randomization.
j) Participant is pregnant, lactating, or has a positive serum ß-subunit human chorionic gonadotropin measured during Screening.
k) Participant has a history or presence of the following clinically relevant cardiovascular conditions:
i) Structural cardiac disease (eg, hypertrophic obstructive cardiomyopathy, unrepaired congenital heart defects). Participants with repaired congenital heart defects should be discussed with the Clinical Trial Physician or designee prior to enrollment.
ii) Cardiac events (eg, myocardial infarction) or diseases that predispose to cardiac complications.
iii) History of stroke, heart failure, or symptomatic bradycardia defined as <5th percentile of normal sinus rhythm HR for age.29
iv) Second degree (Mobitz type II) AV block or higher, sick sinus syndrome, or sino-atrial block.
v) Prolonged QT interval corrected for HR using Fridericia’s formula > 450 msec for both males and females (at either Screening or Day 1 Predose assessment), or is at additional risk for QT interval prolongation (eg, hypokalemia, hypomagnesemia, congenital

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified