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

NCT04852692 | Terminated

• 2 other identifiers: CR10894554179060GVH4001
• Study Type: observational
• Target: 15
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

Chronic Graft vs Host Disease

Keywords

Chronic; Ibrutinib; Retrospective; Prospective

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Part B: Rate of Sustained Response

  At least 5 months (up to 36 weeks)

• Part B: Duration of Response

  Up to 5 months

• Part A and Part B: Corticosteroid Requirement Changes Over Time

  Up to 24 weeks

• Part B: Time to cGVHD Progression

  Up to 36 weeks

• Part B: Change in Lee cGVHD Symptom Scale

  Up to 36 weeks

Study Arms (2)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Janssen Korea, Ltd., Korea: lead

Study Sites (8)

Soonchunhyang University Bucheon Hospital

Bucheon-si, 14584, South Korea

Location

Pusan National University Hospital

Busan, 49241, South Korea

Location

Kyungpook National University Hospital

Daegu, 41944, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Soonchunhyang University Seoul Hospital

Seoul, 04401, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

The Catholic University of Korea Seoul St Mary s Hospital

Seoul, 06591, South Korea

Location

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Study Officials

• Janssen Korea, Ltd., Korea Clinical Trial

  Janssen Korea, Ltd., Korea

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2021
• First Posted: April 21, 2021
• Study Start: June 21, 2021
• Primary Completion: August 6, 2021
• Study Completion: August 6, 2021
• Last Updated: May 23, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

KR (8)