Last Update: Jul 26, 2024
A Single-arm, Multi-center Study of Ruxolitinib for the Treatment of Chinese Patients With Grade II-IV Corticosteroid-refractory Acute Graft Versus Host Disease
ClinicalTrials.gov Identifier:
Novartis Reference Number:CINC424C2416
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 purpose of this study is to assess the efficacy and safety of ruxolitinib therapy in
Chinese adults and adolescents (≥ 12 years old) with Grade II-IV steroid-refractory acute
graft versus host disease (SR-aGvHD). Participants will start with a screening period to assess the eligibility; only
participants who meet all the inclusion and none of the exclusion criteria will start
study treatment from Day 1 to Week 24 or end of treatment. Following safety follow up
visits, participants will receive the long-term follow-up until Month 12.

Steroid-refractory Acute Graft Versus Host Disease
Phase4
Recruiting
54
Jul 04, 2024
Nov 05, 2026
All
12 Years - (Child, Adult, Older Adult)

Interventions

Drug

Ruxolitinib

Ruxolitinib is taken orally daily at 10 mg BID, given as two 5-mg tablets.

Eligibility Criteria

Key Inclusion criteria

- Male or female Chinese participants aged 12 or older at the time of informed
consent. Written informed consent from participant, parent or legal guardian.

- Able to swallow tablets.

- Have undergone alloSCT from any donor source (matched unrelated donor, sibling,
haplo-identical) using bone marrow, peripheral blood stem cells, or cord blood.

- Clinically diagnosed Grades II to IV acute GvHD as per standard criteria occurring
after alloSCT requiring systemic immune suppressive therapy.

- Evident myeloid and platelet engraftment (confirmed within 48 hours prior to study
treatment (ruxolitinib) start):

- Confirmed diagnosis of steroid refractory aGvHD defined as participants administered
high-dose systemic corticosteroids (methylprednisolone 2 mg/kg/day [or equivalent
prednisone dose 2.5 mg/kg/day]), given alone or combined with calcineurin inhibitors
(CNI) and either:

1. Progression based on organ assessment after at least 3 days compared to organ
stage at the time of initiation of high-dose systemic corticosteroid +/- CNI
for the treatment of Grade II to IV aGvHD. OR

2. Failure to achieve at a minimum partial response based on organ assessment
after 7 days compared to organ stage at the time of initiation of high-dose
systemic corticosteroid +/-CNI for the treatment of Grade II to IV. OR

3. Participants who fail corticosteroid taper defined as fulfilling either one of
the following criteria:

- Requirement for an increase in the corticosteroid dose to
methylprednisolone ≥2 mg/kg/day (or equivalent prednisone dose ≥2.5
mg/kg/day). OR

- Failure to taper the methylprednisolone dose to < 0.5 mg/kg/day (or
equivalent prednisone dose <0.6 mg/kg/day) for a minimum of 7 days.

Key Exclusion criteria

- Has received more than one systemic treatment for steroid refractory aGvHD.
Participants who received JAK inhibitor therapy for any indication after initiation
of current alloSCT conditioning.

- Clinical presentation resembling de novo chronic GvHD or GvHD overlap syndrome with
both acute and chronic GvHD features.

- Failed prior alloSCT within the past 6 months. Presence of relapsed primary
malignancy after the alloSCT was performed.

- Presence of an active uncontrolled infection including significant bacterial,
fungal, viral or parasitic infection requiring treatment.

- SR-aGvHD occurring after non-scheduled donor lymphocyte infusion (DLI) administered
for pre-emptive treatment of malignancy recurrence. Note: Participants who have
received a scheduled DLI as part of their transplant procedure and not for
management of malignancy relapse are eligible.

- Presence of significant respiratory disease, severely impaired renal function,
clinically significant or uncontrolled cardiac disease, unresolved cholestatic and
liver disorders (not attributable to aGvHD). Disorders and/or current therapy with
medications that interfere with coagulation or platelet function.

Other protocol-defined inclusion / exclusion criteria may apply

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