Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Study of Investigational Medication in Patients with Persistent or Chronic Immune Thrombocytopenia (ITP)
Study Overview
This was a randomized, double-blind study of rilzabrutinib in patients 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. Patients received rilzabrutinib or placebo 400mg twice daily.
For each patient, the study lasted up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This included 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 was 12 months from the date of the last adult participant to enter the LTE.
For pediatric participants, the maximum duration of the LTE period was 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:
- 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 for adult and Bedside Schwartz Equation for Pediatric participants])
- 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
Had 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
This study investigates persistent or chronic Immune Thrombocytopenia (ITP). The purpose of this study is to evaluate the effects of an investigational medication in people with ITP who have low platelet counts.
Participants will be randomly assigned to receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine. The study involves taking the assigned treatment twice daily and monitoring platelet counts and overall health.
- Who can participate: Participants aged 12 years and older with primary ITP lasting more than 6 months for those under 18, and more than 3 months for adults, may join. They must have had a prior response to certain treatments and meet specific health criteria.
- Study details: Participants will take the investigational medication or a placebo twice daily and attend regular health check-ups. A placebo is an inactive substance that looks like the investigational medicine but does not contain any medicine.
- Study timelines: The study will last up to 60 weeks.
