Graft vs. Host DiseasePhase 4NCT06824103
Study of Efficacy and Safety of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft vs. Host Disease
A Phase 4 study testing Ruxolitinib in people with Graft vs. Host Disease and Chronic Graft vs. Host Disease, run by Novartis Pharmaceuticals at 22 sites in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 12 to 100 years
- Conditions the study is for
- Graft vs. Host Disease, Chronic Graft vs. Host Disease, Corticosteroid-refractory Chronic Graft vs. Host Disease
- What is being tested?
- Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 22 sitesChina
- Study dates
- 9 Sep 2025to 23 May 2031
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
22 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
22 sites in 1 country
China22 sites, 22 recruiting
- Recruiting
Novartis Investigative Site
Beijing, 100034
- Recruiting
Novartis Investigative Site
Beijing, 100070
- Recruiting
Novartis Investigative Site
Changsha, 410000
- Recruiting
Novartis Investigative Site
Chengdu, Sichuan, 610041
- Recruiting
Novartis Investigative Site
Chongqing, 400016
- Recruiting
Novartis Investigative Site
Guangzhou, Guangdong, 510000
- Recruiting
Novartis Investigative Site
Guangzhou, Guangdong, 510515
- Recruiting
Novartis Investigative Site
Guiyang, Guizhou, 550004
- Recruiting
Novartis Investigative Site
Hangzhou, Zhejiang, 310003
- Recruiting
Novartis Investigative Site
Hefei, Anhui, 230001
- Recruiting
Novartis Investigative Site
Nanchang, Jiangxi, 330006
- Recruiting
Novartis Investigative Site
Nanjing, Jiangsu, 210029
- Recruiting
Novartis Investigative Site
Nanning, Guangxi, 530021
- Recruiting
Novartis Investigative Site
Ningbo, Zhejiang, 315016
- Recruiting
Novartis Investigative Site
Shanghai, 200025
- Recruiting
Novartis Investigative Site
Shijiazhuang, Hebei, 050000
- Recruiting
Novartis Investigative Site
Wenzhou, Zhejiang, 325000
- Recruiting
Novartis Investigative Site
Wuhan, Hubei, 430030
- Recruiting
Novartis Investigative Site
Wujiang Nongchang, 065201
- Recruiting
Novartis Investigative Site
Xian, Shanxi, 710061
- Recruiting
Novartis Investigative Site
Xuzhou, Jiangsu, 221003
- Recruiting
Novartis Investigative Site
Zhengzhou, Henan, 450003
About this study
Official title: 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).
Read the 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).
- Conditions
- Graft vs. Host Disease, Chronic Graft vs. Host Disease, Corticosteroid-refractory Chronic Graft vs. Host Disease
- Being tested
- Ruxolitinib
- Keywords
- cGvHD, GvHD, SR-cGvHD, ruxolitinib, INC424, Chinese adult, Pediatric, Corticosteroid, refractory
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called INC424
Ruxolitinib is taken orally daily at 10 mg BID, given as two 5 mg tablets.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib | Experimental | Chinese participants (adult and pediatric) who will receive ruxolitinib daily. Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Cycle 7 Day 1; each Cycle =28 days
Secondary outcomes (8)
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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Cycle 7 Day 1; each cycle = 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2025
- Study start9 Sep 2025
- Primary completion10 May 2029 (estimated)
- Study completion23 May 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft vs. Host DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06824103
- Sponsor's protocol ID
- CINC424D2413
- Status last verified
- Jun 2026
- First submitted
- 4 Feb 2025
- Last updated
- 8 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm Multi-center Study of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft Versus Host Disease After Allogeneic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT06824103
- Acronym / sponsor ID
- CINC424D2413
- 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
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 9 Sep 2025 (actual)
- Primary completion
- 10 May 2029 (estimated)
- Study completion
- 23 May 2031 (estimated)
- First posted
- 13 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib | Experimental | Drug: RuxolitinibChinese participants (adult and pediatric) who will receive ruxolitinib daily. |
Who may qualify
- Ages
- 12 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- 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:
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RuxolitinibAlso: INC424Ruxolitinib is taken orally daily at 10 mg BID, given as two 5 mg tablets. | Drug | Ruxolitinib |
Primary outcome
Overall Response Rate (ORR)
Time frame: Cycle 7 Day 1; each Cycle =28 days
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.
8 secondary outcomes
Failure-free Survival (FFS)
Time frame: up to 3 years
Best Overall Response (BOR)
Time frame: 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
Time frame: end of cycle 3; each cycle = 28 days
Duration of Response (DOR)
Time frame: from first response until cGvHD progression, death, or the date of addition of systemic therapies for cGvHD, approx.36 months
Overall Response (OS)
Time frame: from the date of study treatment (ruxolitinib) initiation to the date of death due to any cause, approx. 36 months
Non-Relapse Mortality (NRM)
Time frame: 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)
Time frame: from date of study treatment to hematologic malignancy relapse/recurrence, approx. 36 months
Reduction of daily corticosteroids dose at cycle 7 day 1
Time frame: Cycle 7 Day 1; each cycle = 28 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
- Individual participant dataSharing planned: Yes. 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.
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Graft vs Host Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
22 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Ruxolitinib in people with Graft vs. Host Disease and Chronic Graft vs. Host Disease, run by Novartis Pharmaceuticals at 22 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: Cycle 7 Day 1; each Cycle =28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2026.