Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis (NCT04624230)
OPEN-LABEL INDUCTION AND MAINTENANCE STUDY OF ORAL CP-690,550 (TOFACITINIB) IN CHILDREN WITH MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital Los Angeles — Los Angeles, California
Age range: 2 Years to 17 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (69 Sites)
Showing the first 25 of 69 sites.
- 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,
- Universitair Ziekenhuis Brussel — Brussels,
- Cliniques Universitaires Saint-Luc — Brussels,
- Stollery Children's Hospital University of Alberta — Edmonton, Alberta
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Ulcerative Colitis
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- Pharmacokinetics of IFX and TNF Concentrations in Serum, Stool, and Colonic Mucosa in Acute Severe Ulcerative Colitis — San Diego, California
- A Study to Evaluate the Safety of Ozanimod Exposure During Pregnancy in Women With Ulcerative Colitis and Their Infants — Eden Prairie, Minnesota
- CP101 for the Treatment of Ulcerative Colitis — Boston, Massachusetts
- A Study on the Safety of TAK-279 and Whether it Can Reduce Inflammation in the Bowel of Participants With Moderately to Severely Active Ulcerative Colitis — Los Angeles, California
- Angiographic Delivery of AD-MSC for Ulcerative Colitis — Rochester, Minnesota
Explore by condition: All Ulcerative Colitis clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.