Study of pharmacokinetics, activity and safety of ruxolitinib in pediatric patients with grade II-IV acute graft vs. host disease

Phase 1

• Conditions: treatment naïve aGvHD grades II-IV or steroid-refractory aGvHD grades II-IV; MedDRA version: 20.0Level: LLTClassification code 10064677Term: Graft versus host disease in intestineSystem Organ Class: 100000004870; MedDRA version: 20.0Level: LLTClassification code 10075161Term: Graft versus host disease in GI tractSystem Organ Class: 100000004870; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2018-000422-55-DK
• Lead Sponsor: ovartis Pharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-10-17
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 43

Inclusion Criteria

• Male or female patients age =28 days and <18 years at the time of informed consent.
• Patients who have undergone alloSCT from any donor source (matched unrelated donor, sibling, haplo-identical) using bone marrow,
peripheral blood stem cells, or cord blood. Recipients of myeloablative or reduced intensity conditioning are eligible.
•Patients with clinically confirmed diagnosis of grades II-IV aGvHD(Harris 2016) within 48 hours prior to study treatment start. Patients may have either: Treatment-naïve aGvHD (criteria per Harris et al. 2016) OR Steroid refractory aGvHD as per institutional criteria, or per physician decision in case institutional criteria are not available and the patient is currently receiving systemic corticosteroids.
• Evident myeloid engraftment with ANC > 1,000/µl and platelet count >20,000/µl. (Use of growth factor supplementation and transfusion support is allowed.)

Other inclusion criteria as per full protocol may apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 45
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

• Has received the following systemic therapy for aGvHD: a)
Treatment-naïve aGvHD patients have received any prior systemic treatment of aGvHD except for a maximum 72h of prior systemic corticosteroid therapy of methylprednisolone or equivalent after the onset of acute GvHD. Patients are allowed to have received prior GvHD prophylaxis which is not counted as systemic treatment (as long as the prophylaxis was started
rior to the diagnsosis of aGvHD); OR b) SR-aGvHD patients have received two or more prior systemic treatments for aGvHD in addition to corticosteroids
• Clinical presentation resembling de novo chronic GvHD or GvHD overlap syndrome with both acute and chronic GvHD features (as defined by Jagasia et al 2015).
• Failed prior alloSCT within the past 6 months.
• Presence of relapsed primary malignancy, or who have been treated for relapse after the alloSCT was performed, or who may require rapid immune suppression withdrawal of immune suppression as pre-emergent treatment of early malignancy relapse.
• Acute GvHD occurring after non-scheduled donor leukocyte infusion (DLI) administered for preemptive
treatment of malignancy recurrence. Note: Patients who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
• Any corticosteroid therapy for indications other than aGvHD at doses > 1 mg/kg/day methylprednisolone (or equivalent prednisone dose 1.25 mg/kg/day) within 7 days of Screening. Routine corticosteroids administered during conditioning or cell infusion is allowed.
• Patients who received JAK
inhibitor therapy for any indication after initiation of current alloSCT conditioning.

Additional exclusion criteria as per full protocol may apply.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified