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.
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.
| Facility | City | Region | Status |
|---|---|---|---|
| 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.
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