Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA 3)
Immune Thrombocytopenia
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia
Eligibility Criteria
Inclusion Criteria:
- Patients will be male and female with primary ITP with duration of >6 months in pediatric participants aged 12 to <18 years (pediatric participants aged 10 to <12 years will be enrolled in the EU [EEA countries] only) and duration of >3 months in ages 18 years and above
- Patients who had a response (achievement of platelet count ≥50,000/µL) to IVIg/anti-D or CSs that was not sustained and who have documented intolerance, insufficient response or any contra-indication to any appropriate courses of standard of care ITP therapy
An average of 2 platelet counts at least 5 days apart of <30,000/µL during the Screening period, and no single platelet count >35,000/µL within 14 days prior to the first dose of study drug
- Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator.
- Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X 10^9/L, AST/ALT ≤1.5 x upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN [unless the patient has documented Gilbert syndrome], glomerular filtration rate >50 [Cockcroft and Gault method])
- Hemoglobin >9 g/dL within 1 week prior to Study Day 1
- All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- Patients must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the patient's guardian and agree to the schedule of assessments
Exclusion Criteria:
- Patients with secondary ITP
- Pregnant or lactating women
- History (within 5 years of Study Day 1) or current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non melanoma skin cancer
- Transfusion with blood, blood products, plasmapheresis, or use of any other rescue medications with intent to increase platelet count within 14 days before Study Day 1
- Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
- Immunosuppressant drugs other than CSs within 5 times the elimination half-life of the drug or 14 days of Study Day 1, whichever is longer
Treatment with rituximab or splenectomy within the 3 months prior to Study Day 1
- Patients treated with rituximab will have normal B-cell counts prior to enrollment
Has received any investigational drug within the 30 days before receiving the first dose of study medication, or at least 5 times elimination half-life of the drug (whichever is longer); patient should not be using an investigational device at the time of dosing
- Patients who previously received treatment with Bruton's Tyrosine Kinase (BTK) inhibitors (except rilzabrutinib) within 30 days before the first dose of study drug are not eligible
- Patients who previously received rilzabrutinib at any time are not eligible
- History of solid organ transplant
- Myelodysplastic syndrome
- Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
- Planned surgery in the time frame of the dosing period
Sites / Locations
- University of Southern California_Investigational Site Number 84024Recruiting
- UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020Recruiting
- Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037Recruiting
- The Oncology Institute of Hope and Innovation_Investigational Site Number 84031Recruiting
- Children's Hospital Colorado_Investigational Site Number 84025Recruiting
- IMMUNOe International Research Centers_Investigational Site Number 84028Recruiting
- ASCLEPES Research Centers_Investigational Site Number 84023Recruiting
- Children's Healthcare of Atlanta_Investigational Site Number 84034Recruiting
- Rush University Medical Center_Investigational Site Number 84029Recruiting
- University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033Recruiting
- Montefiore Medical Center_Investigational Site Number 84032Recruiting
- Cleveland Clinic_Investigational Site Number 84026Recruiting
- The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027Recruiting
- University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035Recruiting
- Investigational Site Number 03206Recruiting
- Investigational Site Number 03205Recruiting
- Investigational Site Number 03209Recruiting
- Investigational Site Number 03208Recruiting
- Investigational Site Number 03609Recruiting
- Investigational Site Number 03606Recruiting
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- Investigational Site Number 03610Recruiting
- Investigational Site Number 03611Recruiting
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- Investigational Site Number 04005Recruiting
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- Investigational Site Number 04001Recruiting
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- Investigational Site Number 04002Recruiting
- Investigational Site Number 07606Recruiting
- Investigational Site Number 07610Recruiting
- Investigational Site Number 07605Recruiting
- Investigational Site Number 07609Recruiting
- Investigational Site Number 07608Recruiting
- Investigational Site Number 07607Recruiting
- Investigational Site Number 07611Recruiting
- CHU Sainte-Justine_Investigational site number 12405Recruiting
- Investigational Site Number 15201Recruiting
- Investigational Site Number 15204Recruiting
- Investigational Site Number 15202Recruiting
- Wuhan Union Hospital of Tongji Medical College of HUST - Investigational Site Number: 15601Recruiting
- Shengjing Hospital of China Medical University - Investigational Site Number: 15603Recruiting
- Shaanxi Provincial People's Hospital - Investigational Site Number: 15607Recruiting
- Qilu Hospital of Shandong University - Investigational Site Number: 15605Recruiting
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences - Investigational Site Number: 15602Recruiting
- Second Affiliated Hospital of Kunming Medical University - Investigational Site Number: 15604Recruiting
- Investigational Site Number 25011Recruiting
- Investigational Site Number 25010Recruiting
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- Investigational Site Number 25008Recruiting
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- Investigational Site Number 27610Recruiting
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- Investigational Site Number 34803Recruiting
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- Investigational Site Number 37605Recruiting
- Investigational Site Number 37606Recruiting
- Investigational Site Number 37607Recruiting
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- Investigational Site Number 38014Recruiting
- Investigational Site Number 38013Recruiting
- Investigational Site Number 38015Recruiting
- Investigational Site Number 38010Recruiting
- Investigational Site Number 38011Recruiting
- Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital - Investigational Site Number: 39203Recruiting
- Ishikawa Prefectural Central Hospital - Investigational Site Number: 39205Recruiting
- Osaka University Hospital - Investigational Site Number: 39202Recruiting
- Kitasato University Hospital - Investigational Site Number: 39207Recruiting
- Local Incorporated Administrative Institution Saitama prefectural hospital organization Saitama prefectural Children's Medical Center - Investigational Site Number: 39206Recruiting
- Saitama Medical University Hospital - Investigational Site Number: 39201Recruiting
- The University of Tokyo Hospital - Investigational Site Number: 39208Recruiting
- Tokyo Metropolitan Bokutoh Hospital - Investigational Site Number: 39209Recruiting
- National Hospital Organization Tokyo Medical Center - Investigational Site Number: 39210Recruiting
- National Center for Child Health and Development - Investigational Site Number: 39204Recruiting
- Investigational Site Number 41004Recruiting
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- Investigational Site Number 41003Recruiting
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- Erasmus MC_Investigational Site Number 52801Recruiting
- Investigational Site Number 57802Recruiting
- Investigational Site Number 57801Recruiting
- Investigational Site Number 61615Recruiting
- Investigational Site Number 61617Recruiting
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- Investigational Site Number 64307Recruiting
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- Investigational Site Number 64301Recruiting
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- Investigational Site Number 64306Recruiting
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- Investigational Site Number 70201Recruiting
- Investigational Site Number 70202Recruiting
- Investigational Site Number 70203Recruiting
- Investigational Site Number 72409Recruiting
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- Investigational Site Number 76405Recruiting
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- Investigational Site Number 79208Recruiting
- Investigational Site Number 79210Recruiting
- Investigational Site Number 79206Recruiting
- Investigational Site Number 79209Recruiting
- Investigational Site Number 80408Recruiting
- Investigational Site Number 80409Recruiting
- Investigational Site Number 80410Recruiting
- Investigational Site Number 82605Recruiting
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Rilzabrutinib
Placebo
Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period
Patients receive matching placebo 400mg orally twice daily for up to 24 weeks