All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks. There will be a 7-day safety follow-up period after receiving the last dose of study medication either in Part A (for those not eligible for Part B or early terminated) or Part B. In addition, each participant will be asked to attend an EOT-Core Part B visit when the last participant completes 52 weeks in Core Part B. The Extended Part B period will last for up to 253 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
Pharmaceutical form: Tablet Route of administration: Oral
The Oncology Institute of Hope & Innovation Site Number : 8400006
Cerritos, California, United States
TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006
Cerritos, California, United States
TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006
Glendale, California, United States
TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006
Long Beach, California, United States
University of Southern California_Investigational Site Number: 8400009
Los Angeles, California, United States
Part A: Proportion of participants with overall hemoglobin response
Response is defined as an increase in hemoglobin (Hb) by ≥2 g/dL from baseline and an absence of transfusion in the last 7 days, without biochemical resolution of hemolysis at the time response is achieved and an absence of rescue medications during the last 4 weeks. Complete Response is defined as hemoglobin ≥11 g/dL (women) or ≥12 g/dL (men), no evidence of hemolysis (normal bilirubin, lactate dehydrogenase (LDH) , haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks.
Time frame: By Week 24 in Part A
Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response
Durable response (Part B) is defined as Hb level ≥10 g/dL with an increase from baseline (Part A) of ≥2 g/dL on three consecutive scheduled visits during Week 24 to Week 50; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
Time frame: By Week 50 in Part B
Proportion of participants with durable hemoglobin response
Durable response (Part A) is defined as Hb level ≥10 g/dL with an increase from baseline of ≥2 g/dL on three consecutive evaluable visits during the 24-week treatment period; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
Time frame: By Week 24 in Part A
Median time from baseline to first hemoglobin response
Time frame: From Day 1 to Week 24 in Part A
Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received
Time frame: After Week 1 of treatment to Week 24 in Part A and Week 305 in Part B
Change from baseline in FACIT-Fatigue scale score
The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale is a 13-item questionnaire used to assesses self-reported fatigue and its impact on daily activities and function.
Time frame: Until Week 24 in Part A and Week 305 in Part B
Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs)
Safety assessment including clinical laboratory evaluations, vital sign measurements and ECG
Time frame: Until Week 24 in Part A and Week 305 in Part B
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006
Santa Ana, California, United States
The Lundquist Institute_Investigational Site Number: 8400005
Torrance, California, United States
TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006
Whittier, California, United States
Georgetown University Hospital_Investigational Site Number: 8400003
Washington D.C., District of Columbia, United States
Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002
Tamarac, Florida, United States
...and 17 more locations