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Steroid-refractory Acute Graft Versus Host DiseasePhase 4NCT06462469

Study of Efficacy and Safety of Ruxolitinib in Patients With Grade II to IV Steroid-refractory Acute Graft vs. Host Disease

A Phase 4 study testing Ruxolitinib in people with Steroid-refractory Acute Graft Versus Host Disease, run by Novartis Pharmaceuticals at 9 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 12 to 100 years
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
36 participants
Where
9 sitesChina
Study dates
4 Jul 2024to 8 Jul 2027
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

  • 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 systemic corticosteroids (methylprednisolone at least 1 mg/kg/day [or equivalent prednisone dose at least 1.25 mg/kg/day]), given alone or combined with calcineurin inhibitors (CNI) and either:
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  • Progression based on organ assessment after at least 3 days compared to organ stage at the time of initiation of systemic corticosteroid +/- CNI for the treatment of Grade II to IV aGvHD. OR
  • 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 systemic corticosteroid +/-CNI for the treatment of Grade II to IV. OR
  • Participants who fail corticosteroid taper defined as fulfilling either one of the following criteria:
  • Requirement for an increase in the corticosteroid dose to methylprednisolone ≥ 1 mg/kg/day (or equivalent prednisone dose ≥ 1.25 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.

Who cannot join

  • 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

Trial locations and contacts

9 sites in 1 country.

9 sites in 1 country

China9 sites

  • Novartis Investigative Site

    Beijing, 100034

  • Novartis Investigative Site

    Beijing, 100070

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510515

  • Novartis Investigative Site

    Hangzhou, Zhejiang, 310003

  • Novartis Investigative Site

    Taian, 271000

  • Novartis Investigative Site

    Tianjin, 300020

  • Novartis Investigative Site

    Wuhan, Hubei, 430030

  • Novartis Investigative Site

    Xian, Shanxi, 710061

  • Novartis Investigative Site

    Zhengzhou, Henan, 450003

Other trials in China

About this study

Official title: 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

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).

Read the detailed description

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.

Being tested
Ruxolitinib
Keywords
SR-aGvHD, aGvHD, acute graft-versus-host disease, ruxolitinib, Chinese patients, corticosteroid-refractory, Grade II-IV, Grade II to IV

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 groups in NCT06462469
Study groupRoleWhat they receive
RuxolitinibExperimentalParticipants will receive ruxolitinib orally of 10 mg BID daily (given as two 5-mg tablets, approximately 12 hours apart).
Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate (ORR) at Day 28 per Investigators

    The ORR at Day 28 defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, mixed response or nonresponse, according to standard criteria and assessed by investigators.

    Measured: Day 28

Secondary outcomes (10)

  1. Durable Overall response rate (ORR) at Day 56

    Durable ORR at Day 56 is defined as the percentage of participants who achieve a complete response (CR) or partial response (PR) at Day 28 and maintain a CR or PR at Day 56.

    Measured: Day 56

  2. Duration of Response (DOR)

    DOR is defined as the time from first response until aGvHD progression or the date of additional systemic therapies for aGvHD.

    Measured: From Week 1 to long term follow up Month 12

  3. Best overall response (BOR)

    Percentage of participants who achieved overall response (complete response (CR) + Partial response (PR) at any time point up to and including Day 28 and before the start of additional systemic therapy for aGvHD.

    Measured: From week 1 to Day 28

  4. Overall survival (OS)

    Overall survival (OS) is defined as the time from the date of start of study treatment to date of death due to any cause.

    Measured: From the date of start of study treatment to date of death, up to approx. 12 months

  5. Non-relapse mortality (NRM)

    Non-relapse mortality (NRM) is defined as the time from date of start of study treatment to date of death not preceded by hematologic disease relapse/progression.

    Measured: From date of start of study treatment to date of death, up to approx. 12 months

  6. Event-free survival (EFS)

    Event-free survival (EFS) is defined as the time from the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death due to any cause.

    Measured: From the date of start of study treatment to the date of hematologic disease relapse/progression, graft failure, or death, up to approx. 12 months

  7. Failure-free survival (FFS)

    Failure-free survival (FFS) is defined as the time from the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment.

    Measured: From the date of start of study treatment to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment, up to approx. 12 months

  8. Malignancy Relapse/Progression (MR)

    Malignancy Relapse/Progression (MR) is defined as the time from date of start of study treatment to hematologic malignancy relapse/progression. Calculated for participants with underlying hematologic malignant disease.

    Measured: From date of start of study treatment to hematologic malignancy relapse/progression, up to approx. 12 months

  9. Reduction of daily corticosteroids dose

    This includes the assessment of systemic corticosteroid use and daily dose, and the percentage of participants successfully tapered off all systemic corticosteroids until Day 56, by time intervals and overall.

    Measured: Up to Day 56

  10. Cumulative incidence of chronic GvHD

    Cumulative incidence of chronic GvHD (cGvHD) includes mild, moderate and severe occurrences.

    Measured: From Week 1 to long term follow up of month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Jun 2024
  2. Study start4 Jul 2024
  3. Primary completion3 Sep 2026
  4. Study completion8 Jul 2027 (estimated)

Resources

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
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06462469
Sponsor's protocol ID
CINC424C2416
Status last verified
Sep 2026
First submitted
5 Jun 2024
Last updated
17 Sep 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.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Ruxolitinib in people with Steroid-refractory Acute Graft Versus Host Disease, run by Novartis Pharmaceuticals at 9 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 11 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (ORR) at Day 28 per Investigators (time frame: Day 28). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06462469.

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 17 Sep 2026.

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