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

NCT06824103 | Recruiting Phase 4

• 1 other identifier: CINC424D2413
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 19

Brief Summary

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).

Conditions

Graft vs. Host Disease; Chronic Graft vs. Host Disease; Corticosteroid-refractory Chronic Graft vs. Host Disease

Keywords

cGvHD; GvHD; SR-cGvHD; ruxolitinib; INC424; Chinese adult; Pediatric; Corticosteroid; refractory

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• Failure-free Survival (FFS)

  up to 3 years

• Best Overall Response (BOR)

  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

  end of cycle 3; each cycle = 28 days

• Duration of Response (DOR)

  from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD, approx.36 months

• Overall Response (OS)

  from the date of study treatment (ruxolitinib) initiation to the date of death due to any cause, approx. 36 months

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

Chinese participants (adult and pediatric) who will receive ruxolitinib daily.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

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

Also known as: INC424

Ruxolitinib

Eligibility Criteria

• Age: 12 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (19)

Novartis Investigative Site

Hefei, Anhui, 230001, China

RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, 510000, China

RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, 510515, China

RECRUITING

Novartis Investigative Site

Nanning, Guangxi, 530021, China

RECRUITING

Novartis Investigative Site

Guiyang, Guizhou, 550004, China

RECRUITING

Novartis Investigative Site

Shijiazhuang, Hebei, 050000, China

RECRUITING

Novartis Investigative Site

Zhengzhou, Henan, 450003, China

RECRUITING

Novartis Investigative Site

Wuhan, Hubei, 430030, China

RECRUITING

Novartis Investigative Site

Nanjing, Jiangsu, 210029, China

RECRUITING

Novartis Investigative Site

Nanchang, Jiangxi, 330006, China

RECRUITING

Novartis Investigative Site

Chengdu, Sichuan, 610041, China

RECRUITING

Novartis Investigative Site

Hangzhou, Zhejiang, 310003, China

RECRUITING

Novartis Investigative Site

Ningbo, Zhejiang, 315016, China

RECRUITING

Novartis Investigative Site

Wenzhou, Zhejiang, 325000, China

RECRUITING

Novartis Investigative Site

Beijing, 100034, China

RECRUITING

Novartis Investigative Site

Beijing, 100070, China

RECRUITING

Novartis Investigative Site

Changsha, 410000, China

RECRUITING

Novartis Investigative Site

Shanghai, 200025, China

RECRUITING

Novartis Investigative Site

Shanhecun, 065201, China

RECRUITING

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2025
• First Posted: February 13, 2025
• Study Start: September 9, 2025
• Primary Completion (Estimated): November 29, 2029
• Study Completion (Estimated): January 11, 2032
• Last Updated: November 26, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

CN (19)