Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Study on Investigational Medication for Chronic Immune Thrombocytopenia Purpura (ITP)
Study Overview
This is a randomized, double-blind study of rilzabrutinib in participants with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Participants will receive rilzabrutinib or placebo 400mg twice daily.
For each participant, the study will last up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This includes Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. Followed by a 4-week post dose follow-up.
For adult participants, the maximum duration of the long-term extension (LTE) period will be 12 months from the date of the last adult participant to enter the LTE.
For pediatric participants, the maximum duration of the LTE period will be 12 months from the date of the last pediatric participant to enter the LTE.
Eligibility Criteria
You may be eligible for this study if you meet the following criteria:
- Conditions: Immune Thrombocytopenia
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Age: 10 years or above
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Gender: All
Inclusion Criteria:
- Participants 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
- Participants 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 contraindication 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 participants 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 ×
10^9/L, AST/ALT ≤1.5 × upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 × ULN [unless the partcipant 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
- Participants must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the participants' guardian and agree to the schedule of assessments.
Exclusion Criteria:
- Participants 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
- Participants 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); participant should not be using an investigational device at the time of dosing
- Participants 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
- Participants 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
This study investigates the use of an investigational medication in people with chronic immune thrombocytopenia (ITP). ITP is a condition where the immune system mistakenly attacks and destroys platelets, which are cells that help blood clot. This can lead to easy or excessive bruising and bleeding. Participants in this study will either receive the investigational medication or a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine.
During the study, participants will take either the investigational medication or placebo twice daily. The study includes different periods: a Blinded Treatment Period where neither the participant nor the doctor knows who is receiving the investigational medication, and an Open-Label Period where everyone knows what medication is being taken. Participants will have regular check-ups and tests to monitor their health and the effects of the treatment.
- Who can participate: Participants are males and females with primary ITP for more than 6 months if they are aged 12 to under 18 years, or more than 3 months if they are 18 years and older. They must have had a response to previous treatments like IVIg or corticosteroids that was not sustained. Adequate blood, liver, and kidney function is required.
- Study details: Participants will take the investigational medication or an investigational placebo twice daily. Participants will undergo regular health check-ups and tests to monitor their condition and the effects of the treatment.
- Study Timelines: The study will last up to 60 weeks.