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Clinical Trials in Sweden / NCT04624230
Active, not recruiting Phase 3

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

NCT04624230 · tracked via the Priya Life Science Sweden tracker
Sponsor
Phase
Phase 3
Started
2021-08-12
Last updated
2026-08-03

Condition(s) studied

Ulcerative Colitis

Investigational drug(s) / intervention(s)

tofacitinib →

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.

Study summary

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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (69)

FacilityCityRegionStatus
Children's Hospital Los Angeles Los Angeles California
University of California, San Francisco Benioff Children's Hospital San Francisco California
University of California, San Francisco Pediatric Clinical Research Center (PCRC) San Francisco California
Connecticut Children's Ambulatory Surgical Center Farmington Connecticut
Connecticut Children's Infusion Center Farmington Connecticut
Connecticut Children's Medical Center Hartford Connecticut
Nicklaus Children's Hospital Miami Florida
Center for Advanced Pediatrics Atlanta Georgia
Children's Healthcare of Atlanta - Arthur M. Blank Hospital Atlanta Georgia
Boston Children's Hospital Boston Massachusetts
Northwell Health - Cohen Children's Medical Center Lake Success New York
Northwell Health - Cohen Children's Medical Center New Hyde Park New York
Cincinnati Children's Hospital Medical Center Cincinnati Ohio
Buerger Center for Advanced Pediatric Care Philadelphia Pennsylvania
Children's Hospital of Philadelphia Philadelphia Pennsylvania
Roberts Center for Pediatric Research Philadelphia Pennsylvania
Texas Children's Hospital - RRO Regulatory (Administrative Offices - Regulatory Location) Houston Texas
Texas Children's Hospital Houston Texas
Seattle Children's Hospital Seattle Washington
The Royal Children's Hospital Parkville Victoria
Universitaire Ziekenhuizen Leuven Leuven Vlaams Brabant
Hôpital Universitaire Des Enfants Reine Fabiola Brussels Belgium
Universitair Ziekenhuis Brussel Brussels Belgium
Cliniques Universitaires Saint-Luc Brussels Belgium
Stollery Children's Hospital University of Alberta Edmonton Alberta
British Columbia Children's Hospital Vancouver British Columbia
IWK Health Centre Halifax Nova Scotia
London Health Sciences Centre - Children's Hospital London Ontario
London Health Sciences Centre - Children's Hospital London Ontario
The Hospital for Sick Children - Division of Gastroenterology, Hepatology and Nutrition Toronto Ontario
CHU Sainte-Justine Montreal Quebec
Tampereen yliopistollinen sairaala Tampere Finland
CHU de Lyon - Hôpital Femme Mère Enfant Bron France
Hôpital Necker Enfants Malades Paris France
Dr. von Haunersches Kinderspital, LMU Munich Bavaria
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Szeged Hungary
Shamir Medical Center (Assaf Harofeh) Be’er Ya‘aqov Israel
Lady Davis Carmel Medical Center Haifa Israel
Shaare Zedek Medical Center Jerusalem Israel
Schneider Children's Medical Center of Israel Petah Tikva Israel

+ 29 more sites — see the full list on the official registry below.

On this site

📄 Xeljanz (tofacitinib) drug profile →

More Pfizer trials in Sweden

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04624230 on ClinicalTrials.gov ↗ ← All trials in Sweden