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Janus Kinase (JAK) Inhibitor

Oral Tofacitinib for Ulcerative Colitis in Children

Phase 3

Recruiting

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

No history of dysplasia or colon cancer

No evidence or history of untreated or inadequately treated active or latent infection with Mycobacterium Tuberculosis

Must not have

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.

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up outcome measured through study completion, an average of 3 and a half years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is designed to see if tofacitinib is effective in treating pediatric UC patients who have either failed or been intolerant to other treatments. The primary objective is to see if the patients go into remission after 44 weeks of treatment.

Who is the study for?

Children aged 2-17 with moderate to severe ulcerative colitis (UC) can join this trial. They must weigh at least 10 kg and have a confirmed UC diagnosis for over 12 weeks, with specific test results showing active disease. Those who've had certain treatments or surgeries recently, live vaccines, or have other health issues like infections or low blood counts cannot participate.

What is being tested?

The trial is testing the drug Tofacitinib in children with UC. It starts with an open-label phase where all participants get Tofacitinib; doses may increase if needed. The main goal is to see if the drug helps achieve remission after 44 weeks of treatment by checking Mayo scores.

What are the potential side effects?

Possible side effects of Tofacitinib include increased risk of infections, changes in blood cell counts, liver enzyme alterations, and potential interactions with other medications that affect immune response or liver function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have never had colon cancer or precancerous colon conditions.

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I do not have an untreated or poorly treated TB infection.

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My ulcerative colitis is moderate to severe, confirmed by a colonoscopy.

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I am between 2 and 17 years old and weigh at least 10 kg.

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I was diagnosed with UC before 6 and tested negative for related genetic disorders.

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My child's UC severity score is 35 or higher.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had major bowel surgery or recent hospital visits for UC.

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I am not taking, nor plan to take, certain strong medications that affect drug metabolism during the study.

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I have not had major surgery or significant trauma in the last 4 weeks.

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I have not taken specific immune system affecting drugs recently.

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I have not had a live vaccine recently nor plan to during the study.

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I regularly use medication to control chronic diarrhea.

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I have taken tofacitinib or a similar medication before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ outcome measured through study completion, an average of 3 and a half years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~outcome measured through study completion, an average of 3 and a half years

This trial's timeline: 3 weeks for screening, Varies for treatment, and outcome measured through study completion, an average of 3 and a half years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Remission by central read Mayo score following 44 weeks in the maintenance phase.

Secondary study objectives

Change from baseline in PUCAI score

Change from baseline in Partial Mayo score

Corticosteroid free remission by Partial Mayo Score

+8 more

Side effects data

From 2020 Phase 4 trial • 4372 Patients • NCT02092467

22%

Upper respiratory tract infection

15%

Bronchitis

13%

Rheumatoid arthritis

13%

Urinary tract infection

12%

Nasopharyngitis

12%

Fall

12%

Herpes zoster

Hypertension

Arthralgia

Diarrhoea

Lymphopenia

Sinusitis

Back pain

Influenza

Osteoarthritis

Latent tuberculosis

Pharyngitis

Nausea

Anaemia

Alanine aminotransferase increased

Cough

Gastroenteritis

Pneumonia

Headache

Coronary artery disease

Femur fracture

Cellulitis

Pulmonary embolism

100%

80%

60%

40%

20%

Study treatment Arm

Tofacitinib 5 mg BID

Tofacitinib 10 mg BID

TNFi

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: tofacitinibExperimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

tofacitinib

2012

Completed Phase 4

~5620

0 / 14Eligibility criteria met