Chronic Graft vs Host Disease
Conditions
Keywords
Chronic, Ibrutinib, Retrospective, Prospective
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.
Interventions
None listed
Sponsors
Janssen Korea, Ltd., Korea
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A and Part B: Percentage of Participants with Overall Response Rate | Week 24 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part B: Rate of Sustained Response | At least 5 months (up to 36 weeks) | Sustained response rate will be assessed. |
| Part B: Duration of Response | Up to 5 months | Duration of response will be assessed. |
| Part A and Part B: Corticosteroid Requirement Changes Over Time | Up to 24 weeks | The change of the corticosteroid dose requirement during the treatment will be assessed. |
| Part B: Time to cGVHD Progression | Up to 36 weeks | 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. |
| Part B: Change in Lee cGVHD Symptom Scale | Up to 36 weeks | Change in Lee cGVHD symptom scale will be assessed. It is a participant reported improvement in symptom burden. |
Countries
South Korea
Outcome results
None listed