The purpose of this study is to assess the safety and clinical efficacy of ibrutinib in subjects with steroid dependent or refractory Chronic Graft Versus Host Disease.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
45
City of Hope Medical Center
Duarte, California, United States
University of California, San Francisco
San Francisco, California, United States
Stanford University
Stanford, California, United States
Phase 1b: To Evaluate the Safety and Tolerability of Ibrutinib in Steroid Dependent/Refractory cGVHD.
Number of participants with dose-limiting toxicities as a measure of safety profile to determine recommended dose of ibrutinib
Time frame: 28 treatment days after last subject enrolled in Phase 1 dose level(s).
Phase 2: Overall Response Rate as the Percentage of Participants With Response
Overall Response Rate is defined as the proportion of subjects who achieved complete response (CR) or partial response (PR). Response criteria are based on NIH cGVHD Response assessment (Pavletic 2006; Measurement of Therapeutic Response, ASBMT Web site).
Time frame: Analysis was conducted with the data extraction date of 15 Sep 2017, with a median follow-up time of 25.56 months.
Sustained Response Rate as the Percentage of Participants With Sustained Response
For subjects who achieved an NIH-defined CR or PR, the proportion of subjects who achieved CR or PR that was sustained for at least 20 weeks (140 days). Intermittent SD was also acceptable.
Time frame: Analysis was conducted with the data extraction date of 15 Sep 2017, with a median follow-up time of 25.56 months.
To Evaluate the Clinical Efficacy of Ibrutinib in Steroid Dependent/Refractory cGVHD by Measuring: Duration of Response (DOR)
For subjects who achieved an NIH-defined CR or PR, the interval between the date of initial documentation of a response and the date of first documented evidence of PD, death, or date of censoring if applicable.
Time frame: Analysis was conducted with the data extraction date of 15 Sep 2017, with a median follow-up time of 25.56 months.
Corticosteroid Requirement Changes Over Time
Average daily corticosteroid dose assessed each week.
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Emory University, Winship Cancer Institute
Atlanta, Georgia, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, United States
Vanderbilt University Medical Center, Henry-Joyce Cancer Clinic
Nashville, Tennessee, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
Time frame: Analysis was conducted with the data extraction date of 15 Sep 2017, with a median follow-up time of 25.56 months.
Percentage of Participants With Overall Improvement in Lee cGVHD Symptom Summary Score
Subject reported improvement in symptom burden. The symptom burden will be measured according to the Lee cGVHD Symptom Scale. A change in \>7 points on the Lee cGVHD Symptom Scale will be considered significant and relates to improvement in quality of life. A score is calculated for each subscale by taking the mean of all items completed if more than 50% were answered and normalizing to a 0 to 100 scale. A total summary score is calculated as the average of these 7 subscales if at least 4 subscales have valid scores. There are 7 subscales (Skin, Energy, Lung, Eye, Nutrition, Mouth and Psychological) with ratings as follow: 0- Not at all, 1- Slightly, 2 Moderately, 3 Quite a bit, 4-Extremely; with a lower values representing a better outcome.
Time frame: Analysis was conducted with the data extraction date of 15 Sep 2017, with a median follow-up time of 25.56 months.
Phase 2b: To Evaluate the Safety and Tolerability of Ibrutinib in Steroid Dependent/Refractory cGVHD
Number of Participants With Adverse Events as a Measure of Safety and Tolerability of Ibrutinib
Time frame: From first dose with study drug until 30 days after the last dose of study drug, up to 36.7 months