Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis

OPEN-LABEL INDUCTION AND MAINTENANCE STUDY OF ORAL CP-690,550 (TOFACITINIB) IN CHILDREN WITH MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS

This study, A3921210 is designed to evaluate the efficacy, safety and pharmacokinetics (PK) of tofacitinib in pediatric participants with moderately to severely active UC. In the US and EU, patients with prior TNFi failure or intolerance will be enrolled. Outside of the US or EU, patients having had inadequate response or intolerance to oral or IV corticosteroids or azathioprine or 6-mercaptopurine or TNFi will be enrolled.

All eligible participants will initially receive open label tofacitinib at a dose expected to produce equivalent systemic exposure to that observed in adults receiving 5 mg BID with the option for individual dose increase to 10 mg BID adult dose equivalent if dose escalation criteria are met.

The primary objective of this study is to evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active UC. The primary endpoint is remission by central read Mayo score following 44 weeks in the maintenance phase. Remission is defined by a Mayo score of 2 points or lower, with no individual subscore exceeding 1 point and a rectal bleeding subscore of 0.

The study Design is an open-label Phase 3 study that includes a screening period of up to 4-weeks duration, an 8-week or 16-week induction phase, a 44-week maintenance phase, and a 24-month extension phase for pediatric participants with moderately to severely active UC. Participants will have a follow-up visit 4 weeks after the last dose of study intervention and a telephone contact 8 weeks later to assess for any adverse events (AEs)/serious adverse events (SAEs). The total maximum duration of this study will be up to 180 weeks.

Study Overview

• Status: Active, not recruiting
• Conditions: Ulcerative Colitis
• Intervention / Treatment: Drug: tofacitinib

• Study Type: Interventional
• Enrollment (Actual): 118
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • The Royal Children's Hospital
• Belgium
  • Brussels, Belgium, 1200
    • Cliniques universitaires Saint-Luc
  • Brussels, Belgium, 1020
    • Hôpital Universitaire Des Enfants Reine Fabiola
  • Brussels, Belgium, 1090
    • Universitair Ziekenhuis Brussel
  • Vlaams Brabant
    • Leuven, Vlaams Brabant, Belgium, 3000
      • Universitaire Ziekenhuizen Leuven
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 1C9
      • Stollery Children's Hospital University of Alberta
  • British Columbia
    • Vancouver, British Columbia, Canada, V6H 3N1
      • British Columbia Children's Hospital
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3K 6R8
      • IWK Health Centre
  • Ontario
    • London, Ontario, Canada, N6A 5W9
      • London Health Sciences Centre - Children's Hospital
    • London, Ontario, Canada, N6C2R5
      • London Health Sciences Centre - Children's Hospital
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children - Division of Gastroenterology, Hepatology and Nutrition
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • CHU Sainte-Justine
• Finland
  • Tampere, Finland, 33520
    • Tampereen yliopistollinen sairaala
• France
  • Bron, France, 69677
    • CHU de Lyon - Hopital Femme Mere Enfant
  • Paris, France, 75015
    • Hôpital Necker Enfants malades
• Germany
  • Bavaria
    • Munich, Bavaria, Germany, 80337
      • Dr. von Haunersches Kinderspital, LMU
• Hungary
  • Szeged, Hungary, 6720
    • Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
• Israel
  • Be’er Ya‘aqov, Israel, 7033001
    • Shamir Medical Center (Assaf Harofeh)
  • Haifa, Israel, 3436212
    • Lady Davis Carmel Medical Center
  • Jerusalem, Israel, 9103102
    • Shaare Zedek Medical Center
  • Petah Tikva, Israel, 4920235
    • Schneider Children's Medical Center of Israel
  • Tel Aviv, Israel, 6423906
    • Tel-Aviv Sourasky Medical Center
• Italy
  • Bologna, Italy, 40133
    • Azienda USL di Bologna - IRCCS ISNB - Programma Gastroenterologia Pediatrica
  • BG
    • Bergamo, BG, Italy, 24127
      • ASST Papa Giovanni XXIII Epatologia e Gastroenterologia Pediatrica e dei Trapianti
  • Naples
    • Naples, Naples, Italy, 80131
      • A.O.U. Federico II
  • RM
    • Roma, RM, Italy, 00161
      • Azienda Ospedaliero - Universitaria Policlinico Umberto I di Roma
• Japan
  • Aichi-ken
    • Obu-shi, Aichi-ken, Japan, 474-8710
      • Aichi Children's Health and Medical Center
  • Fukuoka
    • Kurume-shi, Fukuoka, Japan, 830-0011
      • Kurume University Hospital
  • Gunma
    • Maebashi, Gunma, Japan, 371-8511
      • Gunma University Hospital
  • Miyagi
    • Sendai, Miyagi, Japan, 989-3126
      • Miyagi Children's Hospital
  • Osaka
    • Izumi, Osaka, Japan, 594-1101
      • Osaka Women's and Children's Hospital
    • Takatsuki-shi, Osaka, Japan, 569-8686
      • Osaka Medical and Pharmaceutical University Hospital
  • Saitama
    • Saitama-shi, Saitama, Japan, 330-8777
      • Saitama Prefectural Children's Medical Center
  • Tochigi
    • Shimotsuke, Tochigi, Japan, 329 0498
      • Jichi Medical University Hospital
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8431
      • Juntendo University Hospital
    • Setagaya-ku, Tokyo, Japan, 157-8535
      • National Center for Child Health and Development
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Erasmus Medical Center - Sophia Children's Hospital
  • North Holland
    • Amsterdam, North Holland, Netherlands, 1105 AZ
      • Amsterdam University Medical Center, VUmc Boelelaan
• Poland
  • Lodz, Poland, 93-338
    • Instytut "Centrum Zdrowia Matki Polki"
  • Rzeszów, Poland, 35-302
    • Korczowski Bartosz, Gabinet Lekarski
  • Warsaw, Poland, 04-730
    • Instytut "Pomnik - Centrum Zdrowia Dziecka"
  • Wroclaw, Poland, 50-369
    • Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
  • Masovian Voivodeship
    • Warsaw, Masovian Voivodeship, Poland, 04-501
      • Medical Network Spółka z o.o. WIP Warsaw IBD Point Profesor Kierkuś
• Slovakia
  • Bratislava, Slovakia, 833 40
    • Narodny Ustav Detskych Chorob
• Sweden
  • Gothenburg, Sweden, 416 50
    • Sahlgrenska Universitetssjukhuset
  • Stockholm, Sweden, 118 83
    • Sachsska Children's and Youth Hospital/South General Hospital
  • Stockholm, Sweden, 171 76
    • Karolinska Universitetssjukhuset Barngastroenterologi, hepatologi och nutrition
• United Kingdom
  • London, United Kingdom, E1 1BB
    • Bart's Health NHS Trust
  • Greater London
    • London, Greater London, United Kingdom, SE5 9RS
      • King's College Hospital NHS Foundation Trust
  • WEST Midlands
    • Birmingham, WEST Midlands, United Kingdom, B4 6NH
      • Birmingham Women's and Children's NHS Foundation Trust
• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
    • San Francisco, California, United States, 94158
      • University of California, San Francisco Benioff Children's Hospital
    • San Francisco, California, United States, 94158
      • University of California, San Francisco Pediatric Clinical Research Center (PCRC)
  • Connecticut
    • Farmington, Connecticut, United States, 06032
      • Connecticut Children's Ambulatory Surgical Center
    • Farmington, Connecticut, United States, 06032
      • Connecticut Children's Infusion Center
    • Hartford, Connecticut, United States, 06106
      • Connecticut Children's Medical Center
  • Florida
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Center for Advanced Pediatrics
    • Atlanta, Georgia, United States, 30329
      • Children's Healthcare of Atlanta - Arthur M. Blank Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • New York
    • Lake Success, New York, United States, 11042
      • Northwell Health - Cohen Children's Medical Center
    • New Hyde Park, New York, United States, 11040
      • Northwell Health - Cohen Children's Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
    • Philadelphia, Pennsylvania, United States, 19146
      • Roberts Center for Pediatric Research
    • Philadelphia, Pennsylvania, United States, 19104
      • Buerger Center for Advanced Pediatric Care
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital - RRO Regulatory (Administrative Offices - Regulatory Location)
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Evidence of a personally signed and dated informed consent document and assent document.
• Males and females 2 to less than18 years old and weighing at least 10 kg.
• Having a pathology report that confirms colonic inflammation consistent with UC with a clinical diagnosis of UC for at least 12 weeks prior to baseline, with biopsy report supporting the diagnosis of UC.
• Participants diagnosed with UC at age less than 6 years old, must have had testing and be negative for monogenic disorders associated with very early onset IBD.
• Moderately to severely active UC as defined (via screening colonoscopy) by a Mayo score of at least 6, with a rectal bleeding score of at least 1 and an endoscopic subscore of at least 2.
• Pediatric Ulcerative Colitis Activity Index (PUCAI) score greater or equal to 35 .
• No history of dysplasia or colon cancer.
• No evidence or history of untreated or inadequately treated active or latent infection with Mycobacterium Tuberculosis.

• For participants outside of the United States or the European Union: have had an inadequate response or been intolerant to at least one prior therapy as listed below or have a medical contraindication to such therapies:

  • Oral or intravenous (IV) corticosteroids;
  • Azathioprine or 6-mercaptopurine;
  • TNF inhibitors or anti integrin therapy.
• For participants in the United States and the European Union: have had an inadequate response or intolerance to TNF inhibitors.

• Stable doses of the following therapies for UC:

  • Oral 5 Aminosalicyclic acids (ASA) or sulfasalazine
  • Oral corticosteroids equivalent to prednisone at most 1 mg/kg up to a maximum of 20 mg/day or budesonide up to 9 mg/day.
• female participant is eligible if she is not pregnant or breastfeeding, If she is a woman of child bearing potential, she needs to be using a contraceptive method that is highly effective (with a failure rate of <1% per year).

Exclusion Criteria:

• Diagnosis of indeterminate colitis, isolated proctitis, microscopic colitis, infectious colitis, Crohn's disease, or clinical findings suggestive of Crohn's disease.
• History of symptomatic obstructive intestinal strictures or active ostomy, or history of colectomy, extensive small bowel resection ( greater than100 centimetres) or short bowel syndrome, or hospitalization for UC related reason(s) within 2 weeks of baseline visit.
• Any factors or clinical characteristics potentially related to the risk of venous thromboembolism that may increase the risk associated with study participation or study intervention administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
• Participants who have previously received tofacitinib or another Janus Kinase inhibitor.
• Vaccination or exposure to a live or attenuated vaccine within the 6 weeks prior to the first dose of study drug, or who are expected to be vaccinated or to have household exposure to these vaccines during treatment or during the 6 weeks following discontinuation of study drug.
• Participants having received azathioprine, 6-mercaptopurine, methotrexate, thioguanine, infliximab, adalimumab, golimumab, ustekinumab, interferon, cyclosporine, mycophenolate, tacrolimus, IV or rectally administered corticosteroids, natalizumab, vedolizumab, other antiadhesion molecules, or investigational drugs during the specified time periods prior to baseline whereby they may still have pharmacokinetic and/or pharmacodynamic effect in the body of the participant.
• Previous treatment by leukocyte apheresis including selective lymphocyte, monocyte, or granulocyte apheresis, or plasma exchange within 6 months prior to baseline.
• Treatment by specified prohibited concomitant medications, including moderate to potent CYP3A inducers or inhibitors in the specified time periods prior to the first dose of study drug or are expected to receive any of these medications during the study period.
• Chronic and frequent use of antimotility agents for control of diarrhea (ie, diphenoxylate hydrochloride with atropine sulfate or loperamide).
• History of bowel surgery, including cholecystectomy within 6 months prior to baseline, history of appendectomy within 3 months prior to baseline, or significant trauma or major surgery within 4 weeks of screening visit are excluded.

• Participants with the following laboratory values at screening:

  • Hemoglobin level lower than 9.0 g/Dl.
  • Absolute white blood cell (WBC) count lower than 3000/mm3.
  • Absolute neutrophil count lower than 1200/mm3.
  • Absolute lymphocyte count lower than 750/mm3.
  • Thrombocytopenia as defined by a platelet count lower than 100,000/mm3.
  • Estimated bedside Schwartz Glomerular filtration rate (GFR) lower or equal to 40 mL/min/1.73 m2.
  • Total bilirubin, aspartate aminostransferase (AST) or alanine aminotransferase (ALT) more than 1.5 times the upper limit of normal.
• Positive stool examinations for enteric pathogens, pathogenic ova or parasites, or C. difficile toxin at screening.
• Participants infected with human immunodeficiency virus (HIV) or hepatitis B or C viruses.
• History of more than one episode of HZ, a history of disseminated HZ or disseminated herpes simplex.
• History or current symptoms of any lymphoproliferative disorder (eg, Epstein Barr Virus (EBV) related lymphoproliferative disorder, lymphoma, leukemia, myeloproliferative disorders, multiple myeloma, or signs and symptoms suggestive of currently lymphatic disease).
• Clinically significant infections currently or within 3 months prior to baseline (eg, those requiring hospitalization or parenteral antimicrobial therapy or opportunistic infections), a history of any infection requiring antimicrobial therapy within 2 weeks of baseline, or a history of any infection otherwise judged by the investigator to have the potential for exacerbation by participation in the study.
• Any malignancies or with a history of malignancies, with the exception of adequately treated or excised nonmetastatic basal cell or squamous cell cancer of the skin.
• Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are employees of the Sponsor, including their family members, directly involved in the conduct of the study.
• Participation in other studies involving investigational drug(s) within 2 months prior to study entry and/or during study participation.
• Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or study intervention administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
• Pregnant female participants; breastfeeding female participants; fertile female participants of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and through the telephone follow up visit.
• History of allergies, intolerance or hypersensitivity to lactose or tofacitinib, or any other excipients of the investigational medicinal products, including placebos.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: tofacitinib; Open label tofacitinib 5 mg BID weight based adult equivalent with the option for individual dose increase to 10 mg BID weight based adult equivalent for a limited time if dose escalation criteria are met, prior to returning to 5 mg BID.	Drug: tofacitinib; Open label tofacitinib 5 mg BID weight based adult equivalent with the option for individual dose increase to 10 mg BID weight based adult equivalent for a limited time if dose escalation criteria are met, prior to returning to 5 mg BID.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Remission by central read Mayo score following 44 weeks in the maintenance phase.	Remission is defined by central endoscopy read Mayo score of 2 points or lower, with no individual subscore exceeding 1 point and a rectal bleeding subscore of 0. The primary outcome Mayo score is the summation of 4 subscores as listed below : patient reported stool frequency (scored 0 to 3); patient reported rectal bleeding (scored 0 to 3); central read findings on endoscopy (scored 0 to 3); physician's global assessment (scored 0 to 3) The Mayo score has a scale from 0 to 12 points, with the lower score indicating lower ulcerative colitis (UC) disease activity.	Outcome measured at the end of the 44 weeks of the maintenance phase.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response by Mayo score	Response by Mayo score is defined by a decrease from baseline in Mayo score of at least 3 points and at least 30%, with an accompanying decrease in the subscore for rectal bleeding of at least 1 point or absolute subscore for rectal bleeding of 0 or 1.	Outcome measured at induction Week 8, induction Week 16, and maintenance Week 44
Remission by Mayo score	Remission by Mayo score with local and central endoscopy read (induction Week 8, induction Week 16), and with local endoscopy read only (maintenance week 44).	Outcome measured at induction Week 8, induction Week 16, and maintenance Week 44
Change from baseline in Mayo score.		Outcome measured at induction Week 8, induction Week 16, and maintenance Week 44
Response measured by Partial Mayo Score	Response is defined by a partial Mayo score decrease of 2 points or more from baseline. This score is based on the summation of the following subscores : stool frequency (scored 0 to 3); rectal bleeding (scored 0 to 3); physician global assessment (PGA) (scored 0 to 3) The partial Mayo score has a scale from 0 to 9, with the lower score indicating lower ulcerative colitis (UC) disease activity.	Outcome measured through study completion, an average of 3 and a half years
Change from baseline in Partial Mayo score	Change in partial Mayo score. This score is the summation of 3 distinct dimensions as listed below : stool frequency (scored 0 to 3); rectal bleeding (scored 0 to 3); physician global assessment (PGA) (scored 0 to 3) The partial Mayo score has a scale from 0 to 9, with the lower score indicating lower ulcerative colitis (UC) disease activity.	Outcome measured through study completion, an average of 3 and a half years
Response by PUCAI score	The Pediatric Ulcerative Colitis Activity Index (PUCAI) score is the summation of 6 dimensions that are all reported by the investigator. The PUCAI score varies from 0 to 85 points, with the lower score indicating lower ulcerative colitis (UC) disease activity. The 6 dimensions of the PUCAI score are as follows : abdominal pain; rectal bleeding; stool consistency of most stools; number of stools per 24 hours; nocturnal stools; activity level Response is defined by a PUCAI score decrease of 20 points or more.	Outcome measured through study completion, an average of 3 and a half years
Change from baseline in PUCAI score	The Pediatric Ulcerative Colitis Activity Index (PUCAI) score is the summation of 6 dimensions that are all reported by the investigator; abdominal pain; rectal bleeding; stool consistency of most stools; number of stools per 24 hours; nocturnal stools; activity level The PUCAI score varies from 0 to 85 points, with the lower score indicating lower ulcerative colitis (UC) disease activity.	Outcome measured through study completion, an average of 3 and a half years
Percentage of Participants Achieving Endoscopic Improvement at Week 8, 16, and 44	Endoscopic improvement is defined by Mayo endoscopic sub-score of 0 or 1. The Mayo endoscopic sub-score is used to assess ulcerative colitis activity, and ranges from 0 to 3, and is based on the findings during endoscopy. A lower score is indicative of a lower ulcerative colitis (UC) disease activity.	Outcome measured at induction Week 8, induction Week 16, and maintenance Week 44
Time to flare		Outcome measured from 2 to 4 months in the study, through study completion, an average of 3 and a half years
Change from baseline in fecal calprotectin levels		Outcome measured through study completion, an average of 3 and a half years
Change from baseline in serum high sensitivity C-Reactive Protein (hsCRP) levels		Outcome measured through study completion, an average of 3 and a half years
Corticosteroid free remission by Partial Mayo Score	Remission is defined by a partial Mayo score of 2 points or lower, with no individual subscore exceeding 1 point and rectal bleeding subscore of 0. The subscores of the partial Mayo score are: stool frequency (scored 0 to 3); rectal bleeding (scored 0 to 3); physician global assessment (PGA) (scored 0 to 3)	Outcome measured through study completion, an average of 3 and a half years
Average plasma concentration of tofacitinib (Cavg)		Outcome measured at baseline, induction Week 8, induction Week 16, maintenance Week 16, maintenance Week 44
Evaluation of taste acceptability of tofacitinib oral solution, and acceptability of film coated tablet by choosing one of 5 choices	Taste acceptability will be assessed by asking the participant to select one of 5 choices which most adequately reflects the participant's response to taste. Age appropriate tools (using wording and/or graphic facial expressions) will be used to assess taste acceptability.	Outcome measured at induction week 2
Peak (maximum) plasma concentration of tofacitinib (Cmax)		Outcome measured at baseline, induction Week 8, induction Week 16, maintenance Week 16, maintenance Week 44
Percentage of Participants Achieving Endoscopic Remission at Week 8, 16, and 44	Endoscopic remission is defined by a Mayo endoscopic subscore of 0, out of a maximum of 3 points. The Mayo endoscopic sub-score is used to assess ulcerative colitis activity, and ranges from 0 to 3, and is based on the findings during endoscopy. A lower score is indicative of a lower ulcerative colitis (UC) disease activity.	Outcome measured at induction Week 8, induction Week 16, and maintenance Week 44
Remission by PUCAI score	The Pediatric Ulcerative Colitis Activity Index (PUCAI) score is the summation of 6 dimensions that are all reported by the investigator. The PUCAI score varies from 0 to 85 points, with the lower score indicating lower ulcerative colitis (UC) disease activity. The 6 dimensions of the PUCAI score are as follows : abdominal pain; rectal bleeding; stool consistency of most stools; number of stools per 24 hours; nocturnal stools; activity level Remission is defined by a PUCAI score of less than 10 points.	Outcome measured through study completion, an average of 3 and a half years

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

• Benchimol EI, Bernstein CN, Bitton A, Carroll MW, Singh H, Otley AR, Vutcovici M, El-Matary W, Nguyen GC, Griffiths AM, Mack DR, Jacobson K, Mojaverian N, Tanyingoh D, Cui Y, Nugent ZJ, Coulombe J, Targownik LE, Jones JL, Leddin D, Murthy SK, Kaplan GG. Trends in Epidemiology of Pediatric Inflammatory Bowel Disease in Canada: Distributed Network Analysis of Multiple Population-Based Provincial Health Administrative Databases. Am J Gastroenterol. 2017 Jul;112(7):1120-1134. doi: 10.1038/ajg.2017.97. Epub 2017 Apr 18.
• Konstantinides SV, Meyer G, Becattini C, Bueno H, Geersing GJ, Harjola VP, Huisman MV, Humbert M, Jennings CS, Jimenez D, Kucher N, Lang IM, Lankeit M, Lorusso R, Mazzolai L, Meneveau N, Ni Ainle F, Prandoni P, Pruszczyk P, Righini M, Torbicki A, Van Belle E, Zamorano JL; ESC Scientific Document Group. 2019 ESC Guidelines for the diagnosis and management of acute pulmonary embolism developed in collaboration with the European Respiratory Society (ERS). Eur Heart J. 2020 Jan 21;41(4):543-603. doi: 10.1093/eurheartj/ehz405. No abstract available.
• Kelsen JR, Sullivan KE, Rabizadeh S, Singh N, Snapper S, Elkadri A, Grossman AB. North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition Position Paper on the Evaluation and Management for Patients With Very Early-onset Inflammatory Bowel Disease. J Pediatr Gastroenterol Nutr. 2020 Mar;70(3):389-403. doi: 10.1097/MPG.0000000000002567.
• Turner D, Otley AR, Mack D, Hyams J, de Bruijne J, Uusoue K, Walters TD, Zachos M, Mamula P, Beaton DE, Steinhart AH, Griffiths AM. Development, validation, and evaluation of a pediatric ulcerative colitis activity index: a prospective multicenter study. Gastroenterology. 2007 Aug;133(2):423-32. doi: 10.1053/j.gastro.2007.05.029. Epub 2007 May 21.
• Otley A, Smith C, Nicholas D, Munk M, Avolio J, Sherman PM, Griffiths AM. The IMPACT questionnaire: a valid measure of health-related quality of life in pediatric inflammatory bowel disease. J Pediatr Gastroenterol Nutr. 2002 Oct;35(4):557-63. doi: 10.1097/00005176-200210000-00018.
• Marcovitch L, Nissan A, Mack D, Otley A, Hussey S, Mclean B, Lewis M, Croft N, Barakat FM, Griffiths AM, Turner D. Item Generation and Reduction Toward Developing a Patient-reported Outcome for Pediatric Ulcerative Colitis (TUMMY-UC). J Pediatr Gastroenterol Nutr. 2017 Mar;64(3):373-377. doi: 10.1097/MPG.0000000000001259.
• Taylor SJ, Whincup PH, Hindmarsh PC, Lampe F, Odoki K, Cook DG. Performance of a new pubertal self-assessment questionnaire: a preliminary study. Paediatr Perinat Epidemiol. 2001 Jan;15(1):88-94. doi: 10.1046/j.1365-3016.2001.00317.x.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): August 12, 2021
• Primary Completion (Estimated): March 17, 2027
• Study Completion (Estimated): June 6, 2029

Study Registration Dates

• First Submitted: October 26, 2020
• First Submitted That Met QC Criteria: November 9, 2020
• First Posted (Actual): November 10, 2020

Study Record Updates

• Last Update Posted (Actual): August 3, 2026
• Last Update Submitted That Met QC Criteria: July 31, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric; Ulcerative Colitis; Flare; Tofacitinib Oral; Mayo; PUCAI
• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Colitis; Colitis, Ulcerative; Janus Kinase Inhibitors; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; tofacitinib
• Other Study ID Numbers: A3921210; 2018-002378-30 (EudraCT Number); OVATION (Other Identifier: Alias Study Number); 2023-509694-22-00 (Registry Identifier: CTIS (EU))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No