A Study to Investigate the Comparative Effectiveness of Ibrutinib in Steroid Dependent/Refractory cGVHD Participants

A Retrospective/Prospective, Multicenter, Non-interventional, Historical Control Study Investigating Comparative Effectiveness of IMBRUVICA in Steroid Dependent/Refractory cGVHD Patients

The purpose of this study is to evaluate the effectiveness of ibrutinib compared to conventional salvage treatments in participants with steroid dependent/refractory chronic graft versus host disease (cGVHD) by measuring overall cGVHD response (modified National Institutes of Health [NIH] response defined complete response [CR] and partial response [PR]) at Week 24.

Study Overview

• Status: Terminated
• Conditions: Chronic Graft vs Host Disease

• Study Type: Observational
• Enrollment (Actual): 15

Contacts and Locations

Study Locations

• Korea, Republic of
  • Bucheon-Si, Korea, Republic of, 14584
    • Soonchunhyang University Bucheon Hospital
  • Busan, Korea, Republic of, 49241
    • Pusan National University Hospital
  • Daegu, Korea, Republic of, 41944
    • Kyungpook National University Hospital
  • Seoul, Korea, Republic of, 03080
    • Seoul National University Hospital
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital
  • Seoul, Korea, Republic of, 06351
    • Samsung Medical Center
  • Seoul, Korea, Republic of, 06591
    • The Catholic University of Korea Seoul St. Mary'S Hospital
  • Seoul, Korea, Republic of, 04401
    • Soonchunhyang University Seoul Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with steroid dependent or refractory chronic graft versus host disease (cGVHD) will be included.

Description

Inclusion Criteria:

Part A and Part B

• Must have a confirmed diagnosis of steroid dependent or refractory classic cGVHD defined at any time post-hematopoietic cell transplantation (HCT) as: a) refractory disease - progressive cGVHD manifestations requiring prednisolone at greater than or equal to (>=) 1 milligram per kilogram per day (mg/kg/day) for at least 1 week or persist without improvement despite continued treatment with glucocorticoid (prednisolone at >= 0.5 mg/kg/day or 1 mg/kg every other day) for at least 4 weeks; b) dependent disease: persistent cGVHD manifestations requiring glucocorticoid >= prednisolone 0.25 mg/kg/day or >=0.5mg/kg every other day for at least 8 weeks Part A
• Treated at least 2 or more therapies for cGVHD (including glucocorticoids)
• Participants whose identified last line of conventional salvage treatment is second-fourth line of therapy
• Index date (initiation date of the identified last-line of conventional salvage therapy) at least 28 weeks before the study initiation date Part B
• Treated at least 3 lines therapies for cGVHD (including glucocorticoids)
• Participants who are treated with ibrutinib in their second-fourth line of therapy

Exclusion Criteria:

Part A and Part B

• Known or suspected active acute GVHD
• Received an investigational drug (including investigational vaccines) or used an invasive investigational medical device within 28 days Part A
• Treated with an investigational agent for their identified last-line of conventional salvage therapy
• Pregnant, breastfeeding during last-line of conventional salvage therapy Part B
• Pregnant, breast-feeding, or of childbearing potential without a negative serum or urine pregnancy test within 7 days of enrollment. Male or female participants of childbearing potential unwilling to use effective contraceptive precautions throughout the trial

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Part A: Retrospective Phase; Retrospective collection of data for eligible participants with steroid dependent/refractory chronic graft versus host disease (cGVHD) who initiated salvage treatments from initiation point (administration starting point of the salvage treatment) of the identified last-line of conventional salvage therapy for cGVHD treatment as their second-forth line therapy and will be collected for up to 24 weeks from the initiation point.
Part B: Prospective Phase; Prospectively enroll participants with steroid dependent/refractory cGVHD that are decided to be treated with ibrutinib in second-fourth line therapy for the treatment of cGVHD. Participants will continue to receive corticosteroids as a standard of care.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part A and Part B: Percentage of Participants with Overall Response Rate	Overall response rate is defined as the percentage of participants who achieve complete response (CR) or partial response (PR) according to modified National Institutes of Health (NIH) response.	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part B: Rate of Sustained Response	Sustained response rate will be assessed.	At least 5 months (up to 36 weeks)
Part B: Duration of Response	Duration of response will be assessed.	Up to 5 months
Part A and Part B: Corticosteroid Requirement Changes Over Time	The change of the corticosteroid dose requirement during the treatment will be assessed.	Up to 24 weeks
Part B: Time to cGVHD Progression	Time to chronic graft versus host disease (cGVHD) progression is defined as the time from the first dose of the ibrutinib to modified NIH response defined progression.	Up to 36 weeks
Part B: Change in Lee cGVHD Symptom Scale	Change in Lee cGVHD symptom scale will be assessed. It is a participant reported improvement in symptom burden.	Up to 36 weeks

Collaborators and Investigators

• Sponsor: Janssen Korea, Ltd., Korea
• Investigators: Study Director: Janssen Korea, Ltd., Korea Clinical Trial, Janssen Korea, Ltd., Korea

Study record dates

Study Major Dates

• Study Start (Actual): June 21, 2021
• Primary Completion (Actual): August 6, 2021
• Study Completion (Actual): August 6, 2021

Study Registration Dates

• First Submitted: April 16, 2021
• First Submitted That Met QC Criteria: April 16, 2021
• First Posted (Actual): April 21, 2021

Study Record Updates

• Last Update Posted (Actual): March 29, 2024
• Last Update Submitted That Met QC Criteria: March 28, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Ibrutinib; Chronic; Prospective; Retrospective
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: CR108945; 54179060GVH4001 (Other Identifier: Janssen Korea, Ltd., Korea)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No