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
Sex
ALL
Min age
10 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. 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
2. 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
3. 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.
4. 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 \[Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants\])
5. Hemoglobin \>9 g/dL within 1 week prior to Study Day 1
6. All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
7. 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:
1. Patients with secondary ITP
2. Pregnant or lactating women
3. 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
4. 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
5. Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
6. 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
7. 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
8. 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
9. History of solid organ transplant
10. Myelodysplastic syndrome
11. Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
12. Planned surgery in the time frame of the dosing period
Primary outcome measure(s)
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom — Up to 24 weeks Durable platelet response per guidance in regions except European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for \>=two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, provided that at least 2 non-missing weekly scheduled platelet measurements were at or above 50,000/mcL during the last 6 weeks of the 24-week blinded treatment period.
DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom — Up to 24 weeks Durable platelet response per guidance in European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
Trial sites (155)
Facility
City
Region
Status
University of Southern California_Investigational Site Number 84024
Los Angeles
California
UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020
San Francisco
California
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037
Torrance
California
The Oncology Institute of Hope and Innovation_Investigational Site Number 84031
Whittier
California
Children's Hospital Colorado_Investigational Site Number 84025
Aurora
Colorado
IMMUNOe International Research Centers_Investigational Site Number 84028
Centennial
Colorado
ASCLEPES Research Centers_Investigational Site Number 84023
Weeki Wachee
Florida
Children's Healthcare of Atlanta_Investigational Site Number 84034
Atlanta
Georgia
Rush University Medical Center_Investigational Site Number 84029
Chicago
Illinois
University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033
Louisville
Kentucky
Massachusetts General Hospital Site Number : 84038
Boston
Massachusetts
Montefiore Medical Center_Investigational Site Number 84032
The Bronx
New York
University Hospitals Cleveland Medical Center Site Number : 84036
Cleveland
Ohio
Cleveland Clinic_Investigational Site Number 84026
Cleveland
Ohio
The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027
Philadelphia
Pennsylvania
University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035
Salt Lake City
Utah
University of Washington Medical Centre Site Number : 84041
Seattle
Washington
Investigational Site Number : 3206
Capital Federal
Buenos Aires
Investigational Site Number : 3211
La Plata
Buenos Aires
Investigational Site Number : 3205
Córdoba
Córdoba Province
Investigational Site Number : 3209
Corrientes
Argentina
Investigational Site Number : 3208
San Juan
Argentina
Investigational Site Number : 3607
Kogarah
New South Wales
Investigational Site Number : 3608
Westmead
New South Wales
Investigational Site Number : 3611
Brisbane
Queensland
Investigational Site Number : 3609
Adelaide
South Australia
Investigational Site Number : 3606
Frankston
Victoria
Investigational Site Number : 3610
Perth
Western Australia
Investigational Site Number : 4005
Graz
Austria
Investigational Site Number : 4004
Leoben
Austria
Investigational Site Number : 4001
Linz
Austria
Investigational Site Number : 4003
Steyr
Austria
Investigational Site Number : 4002
Vienna
Austria
Hospital Sao Rafael Instituto D'Or da Bahia Site Number : 7608
Salvador
Estado de Bahia
Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610
Cascavel
Paraná
Hospital De Clinicas De Porto Alegre Site Number : 7605
Porto Alegre
Rio Grande do Sul
Hospital Santa Marcelina Site Number : 7611
São Paulo
São Paulo
CEMEC Oncologica do Brasil Site Number : 7606
Belém
Brazil
HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 7609
Rio de Janeiro
Brazil
Hospital do Servidor Publico Estadual de Sao Paulo Site Number : 7607
São Paulo
Brazil
+ 115 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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