Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
59
Ibrutinib capsule, tablet, or suspension administered orally once daily
Part A- PK (measured by AUC) will be reported descriptively
Time frame: Approximately 24 months
Part B- PK (measured by AUC) will be reported descriptively
Time frame: Approximately 7 years
Number of patients with adverse events as a measure of safety and tolerability
Time frame: Approximately 7 years
Part A- Number of patients with adverse events as a measure of safety and tolerability
Time frame: Approximately 24 months
Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively
Time frame: Approximately 24 months
Part A continuation cohort and Part B-Response rate at 24 weeks
Time frame: Approximately 6 months after last subject in enrolled
Part A continuation cohort and Part B- Duration of response (DOR)
Time frame: Up to 48 weeks
Part A continuation cohort and Part B-Overall survival (OS)
Time frame: Approximately 5 years after last subject enrolled
Part A continuation cohort and Part B-Late Effects Surveillance
Time frame: Up to 5 years post enrollment
Growth Parameter height in meters will be reported descriptively
Subjects will be monitored for growth and development
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City of Hope
Duarte, California, United States
Rady Children's Hospital
San Diego, California, United States
University of California
San Francisco, California, United States
Children's Hospital
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins University
Baltimore, Maryland, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
...and 38 more locations
Time frame: Up to 5 years post enrollment
Growth Parameter weight in kilograms will be reported descriptively.
Subjects will be monitored for growth and development
Time frame: Up to 5 years post enrollment
Available immune reconstitution laboratory parameters will be reported descriptively
Subjects will be monitored for immune reconstitution
Time frame: Up to 5 years post enrollment
Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively
Time frame: Up to 5 years post enrollment