Phase 1/2 study to assess Itacitinib in pediatric subjects with aGVHD
Research type
Research Study
Full title
An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Itacitinib in Combination With Corticosteroids for the Treatment of Steroid-Naïve Acute Graft-Versus-Host Disease in Pediatric Subjects
IRAS ID
256699
Contact name
Robert Wynn
Contact email
Sponsor organisation
Incyte Biosciences UK Ltd
Eudract number
2018-002253-30
Duration of Study in the UK
4 years, 6 months, days
Research summary
Incyte Corporation is sponsoring open-label, single-arm, multicenter, Phase 1/2 study of itacitinib in combination with corticosteroids for the treatment of Grades II to IV of Acute Graft-Versus-Host Disease (aGVHD) in Steroid-Naive pediatric subjects aged from 28 days to < 18 years old.
AGvHD is a serious complication of donor stem cell transplantation. HD is a serious complication of donor stem cell transplantation and remains an important global health burden with thousands of people of all ages affected worldwide. In children, aGVHD represents a major cause of morbidity and mortality after allogeneic stem cell transplant and only about 30% to 50% respond to corticosteroids as initial therapy, and the optimal initial or second-line therapies have not yet been identified.
Itacitinib adipate is a novel, potent, and selective inhibitor of a family of enzymes involved in inflammation called janus kinases (JAK). The aberrant production of these cytokines is associated with the development of a number of conditions, such as aGVHD. Therefore, JAK inhibitors represent potential therapeutic agents for aGVHD.
This study consists of two phases, particularly Phase 1 (safety run-in) where the safety, tolerability, and pharmacokinetics (PK) of itacitinib in combination with corticosteroids is assessed in 5 different cohorts, and Phase 2 (expansion) where the dose found to be safe in Phase 1 is tested in a larger number of pediatric subjects.
Subjects who have received an allogeneic hematopoietic stem cell transplant (allo-HSCT) and have developed Grade II to IV aGVHD may be eligible candidates for treatment per this Protocol. The estimated number of pediatric subjects is 150.
Subject participation is expected to average 12 months. Subjects will receive study treatment until treatment failure (including progression of disease and lack of response), unacceptable toxicity, completion of taper, or death. Transfusion support and continued use of anti-infective medications, GVHD prophylaxis medications, and topical steroid therapies are permitted.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
19/NW/0202
Date of REC Opinion
1 Jul 2019
REC opinion
Favourable Opinion