Drug ID:Drug112
Drug Name:Tofacitinib
CID:9926791
DrugBank ID:DB08895
Modality:Small Molecule
Groups:approved
US Approved:YES
Other Approved:YES
Identifier: NCT00787202, , NCT03281304, , NCT01458574, , NCT01470612, , NCT06625450, , NCT04424303, , NCT05112263, , NCT06095128, , NCT05728008, , NCT05431283, , NCT05313620, , NCT04925973, , NCT06780683, , NCT06274996, , NCT05782907, , NCT06095596, , NCT03653026, , NCT02819635, , NCT03345836, , NCT03006
Molecular Formula:C16H20N6O
Molecular Weight:312.37 g/mol
Isomeric SMILES:C[C@@H]1CCN(C[C@@H]1N(C)C2=NC=NC3=C2C=CN3)C(=O)CC#N
Synonyms:Tofacitinib; 477600-75-2; 3-((3R,4R)-4-methyl-3-(methyl(7H-pyrrolo[2,3-d]pyrimidin-4-yl)amino)piperidin-1-yl)-3-oxopropanenitrile; Cp-690 free base; tofacitinibum; UNII-87LA6FU830; CHEBI:71200; 87LA6FU830; CP-690550 FREE BASE; CP-690,550 FREE BASE
Phase 0: 6
Phase 1: 84
Phase 2: 131
Phase 3: 80
Phase 4: 39
Description:Tofacitinib is an inhibitor of Janus kinases, a group of intracellular enzymes involved in signaling pathways that affect hematopoiesis and immune cell function. It is approved by the FDA for treatment of moderate to severe rheumatoid arthritis that responds inadequately to methotrexate or in those who are intolerant to methotrexate. Besides rheumatoid arthritis, tofacitinib has also been studied in clinical trials for the prevention of organ transplant rejection, and the treatment of psoriasis and ulcerative colitis. Known adverse effects include nausea and headache as well as more serious immunologic and hematological adverse effects. Tofacitinib is marketed under the brand name Xeljanz.

Molecular Structure

2D Molecular Structure

Knowledge Graph

dtIDCIDCompound NameGene IDGene NameSpeciesPubMed IDsAction
dt948 9926791 Tofacitinib 2048 EPHB2 Homo sapiens (human) Inhibitor
dt949 9926791 Tofacitinib 6777 STAT5B Homo sapiens (human) None
dt950 9926791 Tofacitinib 3716 JAK1 Homo sapiens (human) Inhibitor
dt951 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) None
dt952 9926791 Tofacitinib 29110 TBK1 Homo sapiens (human) 25540906 None
dt953 9926791 Tofacitinib 3717 JAK2 Homo sapiens (human) 24417533 Inhibition
dt954 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) Inhibition
dt955 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) 24417533 Inhibition
dt956 9926791 Tofacitinib 3718 JAK3 Homo sapiens (human) 27572962 Inhibition
dt957 9926791 Tofacitinib 3716 JAK1 Homo sapiens (human) 24417533 Inhibition

  • No data available

Phase Distribution
Phase Description
  • Phase 0: Exploratory trials to assess drug behavior in humans
  • Phase 1: Safety trials to determine safe dosage range
  • Phase 2: Efficacy trials to evaluate therapeutic effects
  • Phase 3: Large-scale trials to confirm efficacy and safety
  • Phase 4: Post-marketing surveillance for long-term safety and efficacy

Trial IDTitlePhaseStatusSponsorIndicationsInterventions
NCT04624230 Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis PHASE3 RECRUITING Pfizer Ulcerative Colitis DRUG: tofacitinib Details
NCT01465763 A Study Evaluating The Efficacy And Safety Of CP-690,550 In Patients With Moderate To Severe Ulcerative Colitis PHASE3 COMPLETED Pfizer Ulcerative Colitis DRUG: tofacitinib|DRUG: Placebo Details
NCT04032756 Tofacitinib Registry of Patients With Ulcerative Colitis in Germany None TERMINATED Ced Service GmbH Colitis, Ulcerative|Biologics|Tofacitiniib|Chroni… None Details
NCT01458951 A Study To Evaluate Both The Efficacy and Safety Profile of CP-690,550 In Patients With Moderately to Severely Active Ulcerative Colitis PHASE3 COMPLETED Pfizer Ulcerative Colitis DRUG: tofacitinib|DRUG: Placebo Details
NCT05112263 Tofacitinib Versus Cyclosporine for Steroid Refractory Acute Severe Ulcerative Colitis None NOT_YET_RECRUITING Post Graduate Institute of Medical Education and Research, Chandigarh Ulcerative Colitis DRUG: Tofacitinib|DRUG: Cyclosporine Details
NCT06469424 An Active Surveillance, Post-Authorization Study to Characterize the Safety of Tofacitinib in Patients With Moderately to Severely Active Ulcerative Colitis in the Real-World Setting Using Data From the United Registries for Clinical Assessment and Research (UR-CARE) in the European Union (EU) None RECRUITING Grupo Espanol de Trabajo en Enfermedad de Crohn y Colitis Ulcerosa Ulcerative Colitis None Details
NCT06095128 A Study of Vedolizumab With Tofacitinib in Adults With Ulcerative Colitis (UC) PHASE4 RECRUITING Takeda Ulcerative Colitis DRUG: Vedolizumab|DRUG: Tofacitinib Details
NCT05728008 Retrospective Observational Comparison Study Between Ustekinumab and Tofacitinib as Third Line Therapy in a Multicenter Cohort of Patients With Refractory Ulcerative Colitis. None COMPLETED IRCCS San Raffaele Ulcerative Colitis DRUG: Ustekinumab|DRUG: Tofacitinib Details
NCT04743518 Impact of Anti-TNF, Vedolizumab and Tofacitinib on Aortic Stiffness, Carotid Intima-media Thickness and Cardiovascular Risk of Patients With Ulcerative Colitis None UNKNOWN Centre Hospitalier Universitaire, Amiens Ulcerative Colitis OTHER: Aortic pulse wave velocity|OTHER: carotid … Details
NCT05104723 Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications PHASE1|PHASE2 ENROLLING_BY_INVITATION National Institute of Allergy and Infectious Diseases (NIAID) Chronic Granulomatous Disease|Inflammatory Gastro… DRUG: XELJANZ (tofacitinib) Details
NCT05431283 Real-world Effectiveness of Tofacitinib on Ulcerative Colitis Associated Spondyloarthropathy None UNKNOWN Italian Group for the study of Inflammatory Bowel Disease (IG-IBD) Ulcerative Colitis|Spondyloarthropathy DRUG: Tofacitinib Details
NCT03103412 TD-3504 SAD in Healthy Subjects and Subjects With Ulcerative Colitis (UC) PHASE1 COMPLETED Theravance Biopharma Active Mild Ulcerative Colitis, Active Moderate U… DRUG: TD-3504|DRUG: 15N2-tofacitinib|DRUG: Placebo Details
NCT05069259 Generate Real World Data On Tofacitinib Induction Therapy and Changes In Clinical and Patient Reported Outcomes. None TERMINATED Pfizer Ulcerative Colitis OTHER: Stool sample collection Details
NCT03663400 Identify Predictors That Distinguish Between Tofacitinib Responders and Non-responders Based on Genotype and Cellular and Molecular Profiles From Pinch Biopsies, Blood and Stool Samples None COMPLETED NYU Langone Health Ulcerative Colitis DIAGNOSTIC_TEST: GWAS analysis by Illumina BeadCh… Details
NCT05313620 Effect of Tofacitinib on Coagulation and Platelet Function, and Its Role in Thromboembolic Events PHASE4 RECRUITING Fundación de Investigación Biomédica - Hospital Universitario de La Princesa Ulcerative Colitis|Thromboembolism DRUG: Tofacitinib|DRUG: Infliximab Adalimumab y G… Details
CTRI/2024/01/061649 Prevalence and impact of sarcopenia and frailty in inflammatory bowel disease, and the effect of multi-modal interventions on sarcopenia and clinical outcomes in patients with inflammatory bowel disease - NIL PHASE3 Not Recruiting All India Institute of Medical Sciences Health Condition 1: K509- Crohns disease, unspeci… Intervention1: High protein anti-inflammatory die… Details
CTRI/2023/10/058514 Evaluation of effectiveness and safety of oral tofacitinib in patients with Parthenium dermatitis: A prospective interventional open label study - NIL Not Available Not Recruiting AIIMS, New Delhi Health Condition 1: L237- Allergic contact dermat… Intervention1: Oral Tofacitinib: Tofacitinib is a… Details
ISRCTN96296121 Defining microbial predictors of responsiveness to biologic therapies in Crohn disease and ulcerative colitis Not Available Recruiting Newcastle upon Tyne Hospitals NHS Foundation Trust Crohn disease and ulcerative colitis Digestive S… Current interventions as of 19/12/2024: This is… Details
ISRCTN13495664 SARS-CoV2 Vaccination immunogenicity in Immunosuppressed inflammatory bowel disease Patients Not Available Not Recruiting Imperial College London Immune responses to SARS-CoV-2 vaccination in imm… 600 IBD patients will be recruited stratified acc… Details
CTRI/2021/10/037641 An open label study to compare the efficacy and safety of tofacitinib compared to corticosteroids for inducing remission in moderately severe active ulcerative colitis PHASE4 Recruiting Dayanand Medical College and Hospital Ludhiana Disease.2. Total mayo clinic score <6 or >9. 3.… Health Condition 1: K51- Ulcerative colitis Details

Disease IDDisease NameDefinitionCategoryRelated DrugsMechanism
No data available

Strategy IDTherapeutic StrategySynonymsRelated DrugsMechanism
S07 Blockade of cytokine Cytokine signalling apremilast; roflumilast; filgotinib; tofacitinib; ABT494; ABT-874; ustekinumab; isankizumab; LY-2525623; AMG139; MEDI2079; guselkumab Various T-cell subsets, their differentiation pathways and … Details

The Comparative Risk of Serious Adverse Events With Tofacitinib and TNF Inhibit…

PMID: 35470598
Year: 2022
Relationship Type: Treatment Score: 6.5

Tofacitinib is an oral, small-molecule Janus kinase inhibitor approved in South Korea for the treatment of moderate to severe ulcerative colitis (UC)…

Tofacitinib and newer JAK inhibitors in inflammatory bowel disease-where we are…

PMID: 35462642
Year: 2022
Relationship Type: Treatment Score: 6.5

Inflammatory bowel diseases, comprising ulcerative colitis (UC) and Crohn's disease, are chronic, immune-mediated and progressive inflammatory disord…

Real-world characteristics, treatment experiences and corticosteroid utilisatio…

PMID: 35397501
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib is an oral, small molecule JAK inhibitor for the treatment of UC. We aimed to describe the real-world treatment experience an…

Initial experience of tofacitinib for treating refractory moderate-to-severe ul…

PMID: 35392018
Year: 2022
Relationship Type: Treatment Score: 6.5

Ulcerative colitis (UC) is an incurable, chronic inflammatory disease of the large bowel whose etiology and pathogenesis have not yet been comprehens…

Exposure-Response Characterization of Tofacitinib Efficacy in Moderate to Sever…

PMID: 35380740
Year: 2022
Relationship Type: Treatment Score: 6.5

Tofacitinib is an oral small molecule JAK inhibitor for the treatment of ulcerative colitis. Relationships between plasma tofacitinib concentration a…

Association of C-reactive Protein and Partial Mayo Score With Response to Tofac…

PMID: 35380664
Year: 2023
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib is an oral, small molecule JAK inhibitor for the treatment of ulcerative colitis (UC). These post hoc analyses assessed assoc…

Tofacitinib Appears Well Tolerated and Effective for the Treatment of Patients …

PMID: 35288831
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: Active Crohn's disease increases the risk of strictures, fistulas, and abscesses. Less than 30% of patients with Crohn's disease achieve …

Discovery of a Colon-Targeted Azo Prodrug of Tofacitinib through the Establishm…

PMID: 35275619
Year: 2022
Relationship Type: Treatment Score: 6.5

To mitigate the systemic adverse effects of tofacitinib, 5-ASA-PABA-MAC and 5-ASA-PABA-diamine colon-specific delivery systems were constructed, and …

Effect of tofacitinib in a patient with inflammatory bowel disease-related arth…

PMID: 35260908
Year: 2022
Relationship Type: Treatment Score: 6.5

Extraintestinal manifestations are common in patients with chronic inflammatory bowel disease (IBD). Peripheral arthritis occurs in approximately 10…

Effectiveness of Tofacitinib for Hospitalized Patients with Acute Severe Ulcera…

PMID: 35244825
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: Treatment options for acute severe ulcerative colitis (ASUC) are limited. Tofacitinib, an approved treatment for moderate to severe ulcer…

Tofacitinib combined with leflunomide for treatment of psoriatic arthritis with…

PMID: 35192086
Year: 2022
Relationship Type: Treatment Score: 6.5

Psoriasis is a systemic inflammatory disease that is associated with increased risk of several diseases, such as psoriatic arthritis (PsA), inflammat…

Impact of Biological Therapies and Tofacitinib on Real-world Work Impairment in…

PMID: 35134917
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: There are limited real-world data on the change in total work impairment (TWI) in biological-treated patients with inflammatory bowel dis…

Comparative Risk of Thrombotic and Cardiovascular Events with Tofacitinib and A…

PMID: 35113275
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib and inflammatory bowel disease (IBD) have been associated with increased risks for thromboembolic and cardiovascular events, …

Risk of Infections With Ustekinumab and Tofacitinib Compared to Tumor Necrosis …

PMID: 35066137
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND & AIMS: The comparative safety of therapies is important to inform relative positioning within the therapeutic algorithm. Tumor necrosis f…

Tofacitinib for extraintestinal manifestations of inflammatory bowel disease: A…

PMID: 35063751
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND AND AIMS: Extraintestinal manifestations (EIMs) are commonly seen in patients with inflammatory bowel disease (IBD); management of EIMs is…

Efficacy and safety of tofacitinib in the treatment of ulcerative colitis: real…

PMID: 35000397
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: tofacitinib is a Janus kinase inhibitor approved for the treatment of moderate-severe ulcerative colitis (UC). This study aimed to evalua…

Predictors of Sustained Response With Tofacitinib Therapy in Patients With Ulce…

PMID: 34958359
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib is an oral, small molecule JAK inhibitor for the treatment of ulcerative colitis. We evaluate baseline characteristics as pre…

Treatment of Crohn's Disease and Concomitant Alopecia Areata With Tofacitinib

PMID: 34840997
Year: 2021
Relationship Type: Treatment Score: 6.5

Alopecia areata (AA) is a type of immune-mediated hair loss and is reported in patients with inflammatory bowel disease. This suggests that there mig…

Tofacitinib for a Child with Refractory Steroid-Dependent Ulcerative Colitis: A…

PMID: 34767543
Year: 2021
Relationship Type: Treatment Score: 6.5

BACKGROUND Ulcerative colitis (UC) is a chronic autoimmune inflammatory disease of the colon that infrequently affects children. The disease requires…

Worldwide post-marketing safety surveillance experience with tofacitinib in ulc…

PMID: 34626429
Year: 2022
Relationship Type: Treatment Score: 6.5

BACKGROUND: Tofacitinib is an oral Janus kinase inhibitor for the treatment of ulcerative colitis (UC). Post-marketing surveillance (PMS) is an impor…

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