Clinical trial NCT04852692: A Study to Investigate the Comparative Effectiveness of Ibrutinib in Steroid Dependent/Refractory cGVHD Participants
Official title: A Retrospective/Prospective, Multicenter, Non-interventional, Historical Control Study Investigating Comparative Effectiveness of IMBRUVICA in Steroid Dependent/Refractory cGVHD Patients
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Chronic Graft vs Host Disease
Keywords: Chronic
Ibrutinib
Retrospective
Prospective
Lead sponsor: Janssen Korea, Ltd., Korea
Sponsor class: INDUSTRY
First posted: 2021-04-21
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with steroid dependent or refractory chronic graft versus host disease (cGVHD) will be included.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Part A and Part B: Percentage of Participants with Overall Response Rate
timeFrame: Week 24
Secondary outcomes:
measure: Part B: Rate of Sustained Response
timeFrame: At least 5 months (up to 36 weeks)
measure: Part B: Duration of Response
timeFrame: Up to 5 months
measure: Part A and Part B: Corticosteroid Requirement Changes Over Time
timeFrame: Up to 24 weeks
measure: Part B: Time to cGVHD Progression
timeFrame: Up to 36 weeks
measure: Part B: Change in Lee cGVHD Symptom Scale
timeFrame: Up to 36 weeks
Other outcomes:
Locations:
city: Bucheon-Si
country: Korea, Republic of
facility: Soonchunhyang University Bucheon Hospital
city: Busan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: Seoul
country: Korea, Republic of
facility: Soonchunhyang University Seoul Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: The Catholic University of Korea Seoul St Mary s Hospital
Study officials:
affiliation: Janssen Korea, Ltd., Korea
name: Janssen Korea, Ltd., Korea Clinical Trial
role: STUDY_DIRECTOR
References: