Ruxolitinib as add-on Therapy in Steroid-refractory Graft-vs-host Disease

March 2, 2024 updated by: Yi-Lun Wang

Ruxolitinib Add-on in Steroid-refractory Graft-vs-host Disease After Allogeneic Stem Cell Transplantation: a Single Institutional Experience

Steroid-refractory graft-vs-host disease (SR-GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation (HSCT). We sought to evaluate the effect and safety of ruxolitinib (RUX) add-on in the treatment of patients with SR-GVHD.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Nowadays, second-line treatment for SR-GVHD differs between centers and depends on the physician's experience. The agents commonly used as second and further lines of therapy include anti-thymocyte globulin, mycophenolate mofetil, calcineurin inhibitor, or extracorporeal photopheresis. However, the response varied. This study highlighted that corticosteroid (CS) is not a panacea for treating GVHD, and some patients with remission still experience further relapse. Additionally, the identification of multiple infections adds complexity.

Hope was supposed to come in 2019 after the Food and Drug Administration (FDA) approved RUX for the therapy of SR-GVHD in adult and pediatric patients > 12 years. RUX add-on should be withheld as the risks outweigh the benefits, especially for patients who developed scleroderma and bronchiolitis obliterans. RUX can be added on as needed for the treatment of GVHD, especially in patients receiving DLI as relapse therapy with the conversion from mixed chimerism to complete chimerism. Despite some studies showing the efficacy of RUX across affected organs, some other data bring the opposite results. Thus we conducted this study to reinforce the efficacy of RUX as an add-on therapy for SR-GVHD.

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Taiwan
      • Taoyuan, Taiwan, 333
        • Chang Gung memorial hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 28 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients diagnosed with hematologic disorder

Exclusion Criteria:

  • Those who are not applicable of receiving Ruxolitinib as immunodeficiency-modulation therapy
  • Those who are actively encountering acute infectious illness

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ruxolitinib add-on group
Once diagnosed with steroid-refractory GVHD, after discussion with family, as per their willing, Ruxolitinib will be administered as add-on therapy. Its dose depends on participants' age and body weight. Usually a dosage of 5mg once per day will be applied as initiation and titrated in accordance with clinical response.
Drug: Ruxolitinib
A dosage of 5mg once daily will be applied as initiation. After one week use, dose escalation or de-escalation would depend on clinical response.
Other Names:
  • Jakavi

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
GI symptoms secondary to GVHD
Time Frame: 3 years
Diarrhea event exceeds a frequency of 5 times a day
3 years
Skin symptoms secondary to GVHD
Time Frame: 3 years
Skin rash extends more than 25% of body surface area
3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall survival
Time Frame: 3 years
From diagnosis of SR-GVHD to mortality due to any cause
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Yi-Lun Wang

Investigators

  • Study Director: Tang-Her Jaing, Division of Hematology/Oncology, Department of Pediatrics, Chang Gung Memorial Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2019

Primary Completion (Actual)

January 1, 2020

Study Completion (Actual)

November 30, 2022

Study Registration Dates

First Submitted

November 23, 2022

First Submitted That Met QC Criteria

December 15, 2022

First Posted (Actual)

December 23, 2022

Study Record Updates

Last Update Posted (Actual)

March 5, 2024

Last Update Submitted That Met QC Criteria

March 2, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RSR-1123

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Participants' basic information could be shared. Yet detailed one including morbidity or clinical course may not be exposed.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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