Study of Pharmacokinetics, Activity and Safety of Ruxolitinib in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease

A Phase I/II Open-label, Single-arm, Multi-center Study of Ruxolitinib Added to Corticosteroids in Pediatric Patients With Grade II-IV Acute Graft vs. Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

ClinicalTrials.gov Identifier: NCT03491215

Novartis Reference Number: CINC424F12201

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

The study is an open-label, single-arm, Phase I/II multi-center study to investigate the PK, activity and safety of ruxolitinib added to the patient's immunosuppressive regimen in infants, children, and adolescents ages ≥28 days to <18 years old with either grade II-IV aGvHD or grade II-IV SR-aGvHD. This trial will utilize age groups: Group 1 includes patients ≥12y to <18y, Group 2 includes patients ≥6y to <12y, Group 3 includes patients ≥2y to <6y, and Group 4 includes patients ≥28days to <2y.

Condition 
Acute Graft Versus Host Disease
Phase 
Phase 1
Phase 2
Overall status 
Recruiting
Enrollment count 
55 participants
Start date 
Feb 21, 2019
Completion date 
Aug 07, 2023
Gender 
All
Age(s)
17 Years and older (Child)

Interventions

Drug
Ruxolitinib
Ruxolitinib taken orally (5mg tablets) or oral pediatric formulation and dosage based on age group

Eligibility Criteria

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 a clinically confirmed diagnosis of grades II-IV aGvHD 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, 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.)

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 prior to the diagnosis 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 pre-emptive 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.

Other protocol-defined Inclusion/Exclusion may apply.

Study Locations

Australia
Novartis Investigative Site
Recruiting
Brisbane, 4101
Queensland
Australia
Novartis Investigative Site
Recruiting
Parkville, 3052
Victoria
Australia
Belgium
Novartis Investigative Site
Recruiting
Bruxelles, 1200
-
Belgium
Novartis Investigative Site
Recruiting
Gent, 9000
-
Belgium
Novartis Investigative Site
Recruiting
Laeken, 1020
-
Belgium
Novartis Investigative Site
Recruiting
Leuven, 3000
-
Belgium
Novartis Investigative Site
Recruiting
Liege, 4000
-
Belgium
Canada
Novartis Investigative Site
Recruiting
Toronto, M5G 1X8
Ontario
Canada
Novartis Investigative Site
Recruiting
Montreal, H3T 1C5
Quebec
Canada
Chile
Novartis Investigative Site
Recruiting
Santiago, 664 0166
-
Chile
Denmark
Novartis Investigative Site
Recruiting
Copenhagen, DK-2100
-
Denmark
France
Novartis Investigative Site
Recruiting
Lille, 59000
-
France
Novartis Investigative Site
Recruiting
Nantes Cedex 01, 44093
-
France
Novartis Investigative Site
Recruiting
Paris cedex 15, 75015
-
France
Novartis Investigative Site
Recruiting
Paris Cedex, 75019
-
France
Novartis Investigative Site
Recruiting
Rennes Cedex, 35022
-
France
Novartis Investigative Site
Recruiting
Vandoeuvre Les Nancy, 54511
-
France
Germany
Novartis Investigative Site
Recruiting
Berlin, 13353
-
Germany
Novartis Investigative Site
Recruiting
Duesseldorf, 40225
-
Germany
Novartis Investigative Site
Recruiting
Hamburg, 20246
-
Germany
Novartis Investigative Site
Recruiting
Ulm, 89081
-
Germany
Novartis Investigative Site
Recruiting
Wuerzburg, 97080
-
Germany
Italy
Novartis Investigative Site
Recruiting
Genova, 16147
GE
Italy
Novartis Investigative Site
Recruiting
Roma, 00165
ITA
Italy
Japan
Novartis Investigative Site
Recruiting
Nagoya, 466 8560
Aichi
Japan
Novartis Investigative Site
Recruiting
Saitama, 330 8777
-
Japan
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 03080
-
Korea, Republic of
Novartis Investigative Site
Recruiting
Seoul, 05505
-
Korea, Republic of
Slovenia
Novartis Investigative Site
Recruiting
Ljubljana, 1000
-
Slovenia
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08035
Catalunya
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08025
Cataluña
Spain
Novartis Investigative Site
Recruiting
Madrid, 28009
-
Spain
Novartis Investigative Site
Recruiting
Madrid, 28046
-
Spain
Novartis Investigative Site
Recruiting
Valencia, 46026
-
Spain
Turkey
Novartis Investigative Site
Recruiting
Adana,
-
Turkey

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Name: 
Novartis Pharmaceuticals
Phone: 
+81337978748

Have a question?

Call 1-999-669-6682 or email [email protected]