Study of Efficacy and Safety of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft vs. Host Disease

A Single-arm Multi-center Study of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft Versus Host Disease After Allogeneic Stem Cell Transplantation

The purpose of the study is to assess the efficacy and safety of ruxolitinib in Chinese adult and pediatric participants aged 12 years or older with corticosteroid-refractory chronic graft vs. host disease (SR-cGvHD).

Study Overview

• Status: Recruiting
• Conditions: Chronic Graft vs. Host Disease; Graft vs. Host Disease; Corticosteroid-refractory Chronic Graft vs. Host Disease
• Intervention / Treatment: Drug: Ruxolitinib

Detailed Description

This is a single arm, multi-center, open label study which will enroll approximately 50 participants and investigate the efficacy and safety of ruxolitinib administered in adult and adolescent (≥12 years old) Chinese participants with SR-cGvHD.

The total duration on study for an individual participant will be up to 164 weeks (approximately 3 years).

The study consists of following periods, with each cycle comprised of 4 weeks (28 days):

• Screening Period (Day -28 to Day -1)
• Treatment period (Day 1 to Cycle 39/EOT)
• Safety follow-up (Last dose +30 days)
• Long-term survival follow-up period (EOT to 156 weeks on study).

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Study Locations

• China
  • Beijing, China, 100034
    • Recruiting
    • Novartis Investigative Site
  • Beijing, China, 100070
    • Recruiting
    • Novartis Investigative Site
  • Changsha, China, 410000
    • Recruiting
    • Novartis Investigative Site
  • Chongqing, China, 400016
    • Recruiting
    • Novartis Investigative Site
  • Shanghai, China, 200025
    • Recruiting
    • Novartis Investigative Site
  • Wujiang Nongchang, China, 065201
    • Recruiting
    • Novartis Investigative Site
  • Anhui
    • Hefei, Anhui, China, 230001
      • Recruiting
      • Novartis Investigative Site
  • Guangdong
    • Guangzhou, Guangdong, China, 510515
      • Recruiting
      • Novartis Investigative Site
    • Guangzhou, Guangdong, China, 510000
      • Recruiting
      • Novartis Investigative Site
  • Guangxi
    • Nanning, Guangxi, China, 530021
      • Recruiting
      • Novartis Investigative Site
  • Guizhou
    • Guiyang, Guizhou, China, 550004
      • Recruiting
      • Novartis Investigative Site
  • Hebei
    • Shijiazhuang, Hebei, China, 050000
      • Recruiting
      • Novartis Investigative Site
  • Henan
    • Zhengzhou, Henan, China, 450003
      • Active, not recruiting
      • Novartis Investigative Site
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Recruiting
      • Novartis Investigative Site
  • Jiangsu
    • Nanjing, Jiangsu, China, 210029
      • Recruiting
      • Novartis Investigative Site
    • Xuzhou, Jiangsu, China, 221003
      • Recruiting
      • Novartis Investigative Site
  • Jiangxi
    • Nanchang, Jiangxi, China, 330006
      • Recruiting
      • Novartis Investigative Site
  • Shanxi
    • Xian, Shanxi, China, 710061
      • Recruiting
      • Novartis Investigative Site
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • Recruiting
      • Novartis Investigative Site
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310003
      • Recruiting
      • Novartis Investigative Site
    • Ningbo, Zhejiang, China, 315016
      • Recruiting
      • Novartis Investigative Site
    • Wenzhou, Zhejiang, China, 325000
      • Recruiting
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Signed informed consent must be obtained prior to participation in the study.
• Male or female Chinese participants aged 12 or older at the time of informed consent
• 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. Recipients of non-myeloablative, myeloablative, and reduced intensity conditioning are eligible.

• Evident myeloid and platelet engraftment:

  • Absolute neutrophil count (ANC) >1,000/mm3 AND
  • Platelet count ≥25,000/mm3

Note: Use of growth factor supplementation and transfusion support is allowed during the trial, however, transfusion to reach a minimum platelet count for inclusion is not allowed during screening and at baseline.

• Participants with clinically diagnosed cGvHD staging of moderate to severe according to NIH Consensus Criteria (Jagasia et al 2015) prior to Cycle 1 Day 1.

  • Moderate cGvHD: at least one organ (not lung) with a score of 2, 3 or more organs involved with a score of 1 in each organ, or lung score of 1.
  • Severe cGvHD: at least 1 organ with a score of 3, or lung score of 2 or 3.

• Participants currently receiving systemic corticosteroids for the treatment of cGvHD for a duration of < 12 months prior to Cycle 1 Day 1, and have a confirmed diagnosis of corticosteroid refractory cGvHD defined per 2014 NIH consensus criteria (Martin et al 2015) irrespective of the concomitant use of a calcineurin inhibitor, as follows:

  • A lack of response or disease progression after administration of minimum prednisone 1 mg/kg/day for at least 1 week (or equivalent) OR
  • Disease persistence without improvement despite continued treatment with prednisone at >0.5 mg/kg/day or 1 mg/kg/every other day for at least 4 weeks (or equivalent) OR
  • Increase to prednisone dose to >0.25 mg/kg/day after two unsuccessful attempts to taper the dose (or equivalent)
• Participants has Eastern Cooperative Oncology Group (ECOG) performance status of 0-2

Exclusion Criteria:

For a full list of exclusion criteria, refer to Section 5.2. Key exclusion criteria include

• Participants who have received two or more systemic treatments for cGvHD in addition to corticosteroids ± CNI for cGvHD.
• Participants who have received ROCK2 inhibitors for cGvHD.
• Participants that transition from active aGvHD to cGvHD without tapering off corticosteroids ± CNI and any systemic treatment

Note: Participants receiving up to 30 mg by mouth once a day of hydrocortisone (i.e., physiologic replacement dose) of corticosteroids are allowed.

• Participants who were treated with prior JAK inhibitors for aGvHD; except when the participant achieved complete or partial response and has been off JAK inhibitor treatment for at least 8 weeks prior to Cycle 1 Day 1.
• Failed prior alloSCT within the past 6 months from Cycle 1 Day 1.
• Participants with relapsed primary malignancy, or who have been treated for relapse after the alloSCT was performed.
• SR-cGvHD occurring after a non-scheduled donor lymphocyte infusion (DLI) administered for pre-emptive treatment of malignancy recurrence. Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.

Other protocol-defined inclusion/exclusion may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib; Chinese participants (adult and pediatric) who will receive ruxolitinib daily.	Drug: Ruxolitinib; Ruxolitinib is taken orally daily at 10 mg BID, given as two 5 mg tablets.; Other Names: INC424

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate (ORR)	ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response, according to National Institute of Health (NIH) Consensus Criteria.	Cycle 7 Day 1; each Cycle =28 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Failure-free Survival (FFS)	FFS is defined as the time from the date of start of study treatment to the earliest of: i) relapse or recurrence of underlying disease or death due to underlying disease, ii) nonrelapse mortality, or iii) addition or initiation of another systemic therapy for cGvHD.	up to 3 years
Best Overall Response (BOR)	Percentage of participants who achieved overall response (CR+PR) at any time point (up Cycle 7 Day 1 or the start of additional systemic therapy for cGvHD).	at any point up to cycle 7 day 1 (each cycle is 28 days) or the start of additional systemic therapy for cGvHD, approx. 6 months
ORR at end of cycle 3	Percentage of participants who achieved overall response (CR+PR) at Cycle 4 Day 1.	end of cycle 3; each cycle = 28 days
Duration of Response (DOR)	DOR is assessed for responders only and is defined as the time from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD.	from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD, approx.36 months
Overall Response (OS)	OS is defined as the time from the date of study treatment (ruxolitinib) initiation to the date of death due to any cause.	from the date of study treatment (ruxolitinib) initiation to the date of death due to any cause, approx. 36 months
Non-Relapse Mortality (NRM)	NRM is defined as the time from date of study treatment (ruxolitinib) initiation to date of death not preceded by underlying disease relapse/recurrence.	from date of study treatment (ruxolitinib) initiation to date of death not preceded by underlying disease relapse/recurrence, approx. 36 months
Malignancy Relapse/Recurrence (MR)	Malignancy relapse/recurrence is defined as the time from date of study treatment to hematologic malignancy relapse/recurrence.	from date of study treatment to hematologic malignancy relapse/recurrence, approx. 36 months
Reduction of daily corticosteroids dose at cycle 7 day 1	Systemic corticosteroid use is the percentage of participants with >=50% reduction from baseline in daily corticosteroid dose, the proportion of subjects with reduction from baseline to ≤ 0.2 mg/kg/day methylprednisolone (or equivalent dose of ≤ 0.25 g/kg/day prednisone or prednisolone), and subjects successfully tapered off all systemic corticosteroids at Cycle 7 Day 1, by time intervals and overall.	Cycle 7 Day 1; each cycle = 28 days

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): September 9, 2025
• Primary Completion (Estimated): November 29, 2029
• Study Completion (Estimated): May 23, 2031

Study Registration Dates

• First Submitted: February 4, 2025
• First Submitted That Met QC Criteria: February 10, 2025
• First Posted (Actual): February 13, 2025

Study Record Updates

• Last Update Posted (Actual): May 28, 2026
• Last Update Submitted That Met QC Criteria: May 25, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric; Corticosteroid; refractory; INC424; GvHD; ruxolitinib; cGvHD; Chinese adult; SR-cGvHD
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease; ruxolitinib
• Other Study ID Numbers: CINC424D2413

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No