EUCTR2021-002308-11-PL进行中（未招募）1 期
A phase 2/3, multicenter, randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral ozanimod (RPC1063) in pediatric subjects with moderately to severely active ulcerative colitis with an inadequate response to conventional therapy.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 120
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Age = 2 and < 17 years of age, inclusive, (for the assigned cohort) at the time of informed
- •consent/assent.
- •Note: Subjects with a weight below the 5
- •th percentile for age, should be discussed with
- •the Medical Monitor prior to enrollment.
- •2. One or both parent(s) or legal guardian(s) understands and voluntarily signs informed
- •consent prior to any study-related assessments or procedures (as required by national or
- •local regulations).
- •- Assent from subjects will also be obtained in an age-appropriate manner in
- •conjunction with applicable local regulations. Written assent templates, appropriate
- •for relevant age ranges, will be made available to clinical study sites.
- •3. Willing and able to adhere to the study visit schedule and other protocol requirements
- •including swallowing a capsule (until a sprinkle formulation of ozanimod is available as
- •described in Section 7.1).
- •4. Have had UC diagnosed 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).
- •5. Evidence of UC extending beyond the rectum, as determined by baseline endoscopy
- •(flexible sigmoidoscopy or colonoscopy)
- •6. Has moderately to severely active UC, defined as a 4-component Mayo Score = 6 and
- •= 12 at the time of screening and day 1, including the following: Mayo Endoscopy Subscore = 2,
- •Rectal Bleeding Score = 1, and Stool Frequency Score = 1.
- •7. Has had an inadequate response, loss of response to, or is intolerant to at least 1 of the
- •following treatments for UC (Appendix B):
- •a. oral aminosalicylates (eg, mesalamine, sulfasalazine, olsalazine, balsalazide) (N/A in France)
- •b. systemic corticosteroids (eg, oral prednisone, budesonide MMX, IV corticosteroids)
- •c. immunomodulators (eg, AZA, 6-MP, cyclosporine, MTX)
- •d. biologic therapy (eg, abatacept, infliximab, etanercept, adalimumab, anakinra,
- •rituximab, vedolizumab, and golimumab)
- •e. other systemic immunomodulatory treatments for UC, such as tofacitinib
- •Note: criterion 7b-7e are N/A in Germany
- •8. Subjects may concurrently receive treatment with oral 5-ASAs, or with prednisone or
- •equivalent (= 0.5 mg/kg/ day up to 20 mg/day) or beclomethasone < 5mg/day
- •- If on oral 5-ASAs, the dose must have been stable for at least 3 weeks prior to
- •screening endoscopy = baseline .
- •- If on corticosteroids, the dose must have been stable for at least 2 weeks prior to
- •screening endoscopy and must remain stable through the first 5 weeks of treatment
- •(ie, until the Week 5 study visit).
- •-If discontinuing corticosteroids (e.g. MMX), subject must taper as described in Section 8.1.1.
- •9. Inclusion criterion 9 is not applicable for protocol amendment 2.0.
- •10. Must have documentation of vaccinations per standard immunization schedule including
- •complete Varicella vaccination at least 30 days prior to randomization. (VZV and IgG +ve)
- •11. Females of childbearing potential (FCBP)1 must agree to practice a highly effective
- •method of contraception2
- •throughout the study until completion of the 90-day Safety
- •Follow-up Visit. Highly effective methods of contraception are those that alone or in
- •combination result in a failure rate of a Pearl index of less than 1% per year when used
- •consistently and correctly. Selection of contraceptive methods should be based on those
- •available/approved as per national or local practice. Acceptable methods of bir
排除标准
- •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 subject at risk by participating in the study
- •Crohn’s disease, indeterminate colitis or the presence or history of a fistula consistent with Crohn’s disease or microscopic colitis, radiation colitis or ischemic colitis
- •+ve for toxin producing Clostridium difficile or PCR exam of stool. If +ve, subjects may be rescreened after appropriate treatment and retested no earlier than 7 days after treatment completion
- •+ve examination for pathogens. If positive, subjects may be treated and rescreened
- •History of treatment with topical rectal 5-aminosalicylic acid or topical rectal steroids within 2 weeks of Screening endoscopy or anti-motility medications
- •Apheresis within 2 weeks of randomization
- •Delayed growth or pubertal development and who will not maintain a stable dose of corticosteroids through Week 5
- •Pregnancy (also on day 1), lactation, or positive serum ß-hCG during Screening
- •History or presence of: Structural cardiac disease, Cardiac events or diseases that predispose to cardiac complications, Prolonged QT interval corrected for heart rate using Fridericia’s formula>450 msec for both genders, or additional risk for QT interval prolongation, resting HR < 55 bpm in subjects 12-17 years old, resting HR < 65 bpm in subjects 6-11 years old, or resting HR < 75 bpm in subjects 2-5 years old. Subjects allowed one recheck per Visit, untreated sleep apnea
- •History of DM type 1, or uncontrolled DM type 2 glycosylated hemoglobin (HbA1c > 9%) or is a diabetic subject with significant comorbid conditions
- •History of uveitis within the last year prior or at Screening, or history or evidence of retinal disease
- •Subject has any known active bacterial, viral, fungal, mycobacterial infection, or any major episode infection that required hospitalization or treatment with intravenous antibiotics within 30 days of Screening or oral antibiotics within 14 days of screening
- •In the case of prior SARS-CoV-2 infection, symptoms must be resolved and based on Investigator assessment, there are no sequelae that would place the subject at a higher risk of receiving investigational treatment
- •Recurrent or chronic infection, recurrent urinary tract infections are allowed, there should also be documented evidence of surveillance for dysplasia for all subjects with left-sided colitis of > 12 years’ duration and total or extensive colitis of > 8 years’ duration.
- •History of cancer
- •History of or currently active primary or secondary immunodeficiency, or subjects with known genetic disorders as a cause for colitis
- •ECG showing clinically significant abnormality
- •Serum creatinine > 1.4 mg/dL for females, or > 1.6 mg/dL for males
- •Liver function impairment; or, persisting elevations of alanine aminotransferase or aspartate aminotransferase > 2x upper limit of normal; or direct bilirubin > 1.5x UL
- •Platelet < 100,000/µL
- •Hemoglobin 7 < 8 g/dL
- •Neutrophil < 1500/µL
- •Absolute white blood < 3500/µL
- •ALC < 800/µL
- •Stool positive for pathogens
- •Pulmonary function test measurements: Forced expiratory volume at 1 second or a forced vital capacity < 70% of predicted values, Hypersensitivity to active ingredients or excipients of ozanimod
- •Treated with biological or investigational agent, including for SARS-CoV-2, within 4 weeks of that agent prior to the first dose of IP. Undetect
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