Ruxolitinib for corticosteroid-refractory chronic graft-versus-host disease in Chinese patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single-arm, open-label, multi-center Phase 4 trial enrolling about 50 Chinese participants aged 12 years and older with moderate-to-severe corticosteroid-refractory chronic graft-versus-host disease after allogeneic stem cell transplant. Participants will receive oral ruxolitinib with treatment cycles of 28 days and will be followed on study for up to 164 weeks including safety and long-term survival follow-up. The trial requires baseline neutrophil and platelet thresholds and allows growth factor or transfusion support during treatment but not to meet screening limits. Outcomes collected include measures of cGvHD response, safety, and long-term survival.

Why it matters

Who can join: the full rules

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.

Where it's running

• Novartis Investigative Site
  Hefei, Anhui, China Recruiting
• Novartis Investigative Site
  Guangzhou, Guangdong, China Recruiting
• Novartis Investigative Site
  Guangzhou, Guangdong, China Recruiting
• Novartis Investigative Site
  Nanning, Guangxi, China Recruiting
• Novartis Investigative Site
  Guiyang, Guizhou, China Recruiting
• Novartis Investigative Site
  Shijiazhuang, Hebei, China Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Using Ruxolitinib to prevent complications after haploidentical stem cell transplants

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  We are testing whether combining LBP-MARCO with ruxolitinib is safe for adults with GVHD after a stem cell transplant.

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• DMR-001 in CALR-mutant myeloproliferative neoplasms

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• Testing WJ01024 Alone and with Ruxolitinib for Myelofibrosis with Splenomegaly

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  Recruiting Phase 1 Henan Cancer Hospital · Zhengzhou, Henan · NCT07802405

• Belumosudil vs. Ruxolitinib for hepatic cGVHD after HSCT

  This trial tests whether belumosudil or ruxolitinib works better for hepatic chronic GVHD after a stem cell transplant in people 12 and older.

  Recruiting First Affiliated Hospital of Zhejiang University · Hangzhou, Zhejiang · NCT07794514

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.