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

NCT05663827 | Completed Phase 3

• 1 other identifier: RSR-1123
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Steroid Refractory GVHD

Keywords

acute lymphoblastic leukemia; hematopoietic stem cell transplantation

Outcome Measures

Primary Outcomes (2)

• GI symptoms secondary to GVHD

  Diarrhea event exceeds a frequency of 5 times a day

  3 years

• Skin symptoms secondary to GVHD

  Skin rash extends more than 25% of body surface area

  3 years

Secondary Outcomes (1)

• Overall survival

  3 years

Study Arms (1)

Ruxolitinib add-on group

EXPERIMENTAL

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

Interventions

Ruxolitinib DRUG

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.

Also known as: Jakavi

Ruxolitinib add-on group

Eligibility Criteria

• Age: 3 Months - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients diagnosed with hematologic disorder

You may not qualify if:

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

Sponsors & Collaborators

• Yi-Lun Wang: lead

Study Sites (1)

Chang Gung Memorial Hospital

Taoyuan District, 333, Taiwan

Location

Related Publications (3)

• Malard F, Huang XJ, Sim JPY. Treatment and unmet needs in steroid-refractory acute graft-versus-host disease. Leukemia. 2020 May;34(5):1229-1240. doi: 10.1038/s41375-020-0804-2. Epub 2020 Apr 3.

  PMID: 32242050 RESULT

• Wolff D, Fatobene G, Rocha V, Kroger N, Flowers ME. Steroid-refractory chronic graft-versus-host disease: treatment options and patient management. Bone Marrow Transplant. 2021 Sep;56(9):2079-2087. doi: 10.1038/s41409-021-01389-5. Epub 2021 Jul 3.

  PMID: 34218265 RESULT

• Przepiorka D, Luo L, Subramaniam S, Qiu J, Gudi R, Cunningham LC, Nie L, Leong R, Ma L, Sheth C, Deisseroth A, Goldberg KB, Blumenthal GM, Pazdur R. FDA Approval Summary: Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease. Oncologist. 2020 Feb;25(2):e328-e334. doi: 10.1634/theoncologist.2019-0627. Epub 2019 Oct 22.

  PMID: 32043777 RESULT

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Tang-Her Jaing

  Division of Hematology/Oncology, Department of Pediatrics, Chang Gung Memorial Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal investigator, Division of Hematology/Oncology, Department of Pediatrics, Chang Gung Memorial Hospital

Model Details: Cases with steroid-refractory GVHD would be given an add-on therapy of age/weight adjusted dosage of Ruxolitinib

Study Record Dates

• First Submitted: November 23, 2022
• First Posted: December 23, 2022
• Study Start: January 1, 2019
• Primary Completion: January 1, 2020
• Study Completion: November 30, 2022
• Last Updated: March 5, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1)