NCT03774082

Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant

A Phase II Open-label, Single-arm, Multi-center Study of Ruxolitinib Added to Corticosteroids in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplantation

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 28 Days – 17 Years
• Healthy volunteers: Not accepted

Summary

This open-label, single-arm, Phase II multi-center study enrolled 46 participants and investigated the activity, pharmacokinetics and safety of ruxolitinib added to the subject's immunosuppressive regimen among infants, children, and adolescents aged ≥28 days to \<18 years old with either moderate to severe treatment-naive cGvHD or SR-cGvHD. Although 46 participants were enrolled,1 participant (enrolled in the ≥6y to \<12y age group) received study treatment beyond protocol requirements and was excluded from analyses.

Detailed description

Subjects were grouped according to their age as follows: * Group 1 included subjects ≥12y to \<18y * Group 2 included subjects ≥6y to \<12y * Group 3 included subjects ≥2y to \<6y and * Group 4 included subjects ≥28days to \<2y. Enrollment initiation into the youngest age group, Group 4, was subject to the availability of data in this age group from another study, as well as a review of available PK, safety, and activity data generated from Groups 1 to 3 in the current study. At least 5 evaluable participants per group were needed for the primary analysis in Groups 1, 2 and 3. No minimum number of evaluable participants were needed in Group 4. Enrollment was completed prior to the availability of the data and so no subjects were enrolled in Group 4. After a screening period of Day -28 to Day -1: eligible subjects started study treatment on Cycle 1 Day 1 and were treated for up to a maximum of 3 years (39 cycles/156 weeks) or until early discontinuation. Subjects who discontinued study treatment for any reason earlier than 39 cycles were followed every 6 months until 3 years from their first dose of study treatment was reached.

Conditions

• Graft vs Host Disease

Interventions

Type	Name	Description
DRUG	INC424	Ruxolitinib was taken orally based on age groups as follows: Group 1 (\>=12y to \<18y): 10mg bid as tablet Group 2 (\>=6y to \<12y): 5mg bid as tablet or liquid Group 3 (\>=2y to \<6y): 4mg/m2 bid as liquid

Timeline

• Start date: 2020-05-20
• Primary completion: 2022-02-25
• Completion: 2024-08-26
• First posted: 2018-12-12
• Last updated: 2025-12-11
• Results posted: 2025-04-17

Locations

21 sites across 14 countries: Brazil, Canada, Czechia, India, Italy, Japan, Russia, Slovakia, South Korea, Spain, Switzerland, Taiwan, Thailand, Turkey (Türkiye)