Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Throm… (NCT04562766) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
United States, Argentina, Australia232 participantsStarted 2020-12-14
Plain-language summary
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.
Who can participate
Age range
10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
. 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 \[Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants\])
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom
Timeframe: Up to 24 weeks
2
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom
. 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
. 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