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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04562766




Registration number
NCT04562766
Ethics application status
Date submitted
18/09/2020
Date registered
24/09/2020
Date last updated
28/05/2024

Titles & IDs
Public title
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Scientific title
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Secondary ID [1] 0 0
PRN1008-018
Secondary ID [2] 0 0
EFC17093
Universal Trial Number (UTN)
Trial acronym
LUNA 3
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immune Thrombocytopenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Rilzabrutinib
Treatment: Drugs - Placebo

Experimental: Rilzabrutinib - Participants receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period

Placebo Comparator: Placebo - Participants receive matching placebo 400mg orally twice daily for up to 24 weeks


Treatment: Drugs: Rilzabrutinib
400mg Caplet

Treatment: Drugs: Placebo
400mg Caplet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Durable platelet response during the last 6 weeks of the 24-week blinded treatment period (not for EU and UK)
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
for EU and UK: Proportion of adult participants able to achieve platelet counts at or above 50,000/µL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy
Timepoint [2] 0 0
24 weeks
Secondary outcome [1] 0 0
Number of weeks with platelet count =50,000/µL OR between =30,000/µL and <50,000/µL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Number of weeks with platelet counts =30,000/µL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Time to first platelet count of =50,000/µL OR between =30,000/µL and <50,000/µL and doubled from baseline
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Proportion of participants requiring rescue therapy during the 24-week blinded treatment period
Timepoint [4] 0 0
24 weeks
Secondary outcome [5] 0 0
Change from baseline on Item 10 of the ITP-Patient Assessment Questionnaire in adult participants (=18 years) at Week 13
Timepoint [5] 0 0
From baseline to Week 13
Secondary outcome [6] 0 0
for EU and UK: Change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25
Timepoint [6] 0 0
At Week 25
Secondary outcome [7] 0 0
Proportion of participants able to achieve stable platelet response, within a period of 24 weeks following initial achievement of the platelet response
Timepoint [7] 0 0
24 weeks
Secondary outcome [8] 0 0
Frequency and severity of Treatment Emergent Adverse Events
Timepoint [8] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Secondary outcome [9] 0 0
Frequency and severity of bleeding TEAEs
Timepoint [9] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Secondary outcome [10] 0 0
Plasma concentrations of rilzabrutinib
Timepoint [10] 0 0
Until 52 weeks
Secondary outcome [11] 0 0
Change from baseline on the Symptoms, Bother and Activity domains of the ITP Patient Assessment Questionnaire (ITP-PAQ) in adult participants (=18 years)
Timepoint [11] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Secondary outcome [12] 0 0
Change from baseline in disease-specific QoL as measured by the Kids' ITP Tools (ITP-KIT) score in pediatric participants
Timepoint [12] 0 0
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose

Eligibility
Key inclusion criteria
1. 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

2. 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

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 participants 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 ×
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])

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. 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.
Minimum age
10 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Participants 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

- Participants treated with rituximab will have normal B-cell counts prior to
enrollment

8. 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

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
20/11/2026
Actual
Sample size
Target
194
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 3607 - Kogarah
Recruitment hospital [2] 0 0
Investigational Site Number : 3608 - Westmead
Recruitment hospital [3] 0 0
Investigational Site Number : 3611 - Brisbane
Recruitment hospital [4] 0 0
Investigational Site Number : 3609 - Adelaide
Recruitment hospital [5] 0 0
Investigational Site Number : 3606 - Frankston
Recruitment hospital [6] 0 0
Investigational Site Number : 3610 - Perth
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4101 - Brisbane
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
3199 - Frankston
Recruitment postcode(s) [6] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
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California
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United States of America
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Colorado
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Florida
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United States of America
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Georgia
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Illinois
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Kentucky
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Massachusetts
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New York
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Ohio
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Pennsylvania
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Utah
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Washington
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Argentina
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Buenos Aires
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Argentina
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Córdoba
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Argentina
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Corrientes
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Argentina
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San Juan
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Austria
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Graz
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Austria
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Leoben
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Austria
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Linz
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Austria
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Steyr
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Austria
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Wien
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Brazil
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Bahia
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Brazil
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Paraná
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Brazil
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Rio Grande Do Sul
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Brazil
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São Paulo
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Belem Do Para
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Rio de Janeiro
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Canada
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Alberta
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Ontario
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Canada
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Quebec
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Chile
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Coquimbo
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Chile
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Reg Metropolitana De Santiago
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Chile
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Valparaíso
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China
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Hubei
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China
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Liaoning
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Shaanxi
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Shandong
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Tianjin
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Yunnan
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Hangzhou
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China
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Hefei
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Nanchang
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Tangshan
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China
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Wuxi
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ZhenJiang
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Angers
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Creteil
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Nantes Cedex 1
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Düsseldorf
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Germany
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Frankfurt am Main
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Germany
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Recklinghausen
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Hungary
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Budapest
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Hungary
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Debrecen
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Gyor
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Hungary
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Nyíregyháza
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Hungary
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Szekesfehervar
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Israel
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Haifa
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Israel
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Kfar Saba
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Israel
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Tel Aviv
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Israel
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Tel Hashomer
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Israel
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Zerifin
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Toscana
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Bologna
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Milano
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Trieste
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Vicenza
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Ibaraki
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Ishikawa
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Kanagawa
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Osaka
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Saitama
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Chiba
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Hiroshima City
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Saitama-shi
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Busan-gwangyeoksi
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Gyeonggi-do
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Korea, Republic of
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Seoul-teukbyeolsi
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Mexico
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Nuevo León
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Mexico
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Chihuahua
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Mexico
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Ciudad de México
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Mexico
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Delegacion Benito Juarez
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Mexico
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Durango
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Mexico
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Zapopan
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Netherlands
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Rotterdam
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Norway
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Bergen
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Norway
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Gralum
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Poland
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Pomorskie
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Poland
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Wielkopolskie
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Gdynia
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Poland
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Poznan
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Warszawa
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Poland
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Wroclaw
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Russian Federation
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Moscow
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Russian Federation
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Novosibirsk
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Russian Federation
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Pyatigorsk
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Russian Federation
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Samara
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Russian Federation
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St. Petersburg
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Russian Federation
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Tula
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Singapore
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Singapore
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Spain
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Málaga
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Spain
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Valenciana, Comunidad
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Spain
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Barcelona
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Spain
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Burgos
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Spain
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Madrid
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Spain
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Murcia
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Spain
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Sevilla
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Thailand
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Bangkok
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Thailand
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Chiangmai
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Thailand
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Khon Kaen
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Thailand
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Songkla
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Turkey
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Ankara
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Turkey
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Istanbul
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Turkey
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Izmir
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Turkey
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Kayseri
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Ukraine
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Dnipropetrovsk
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Ukraine
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Kryvyi Rih City
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Ukraine
State/province [122] 0 0
Kyiv
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United Kingdom
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London, City Of
Country [124] 0 0
United Kingdom
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Harrow
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United Kingdom
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London
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United Kingdom
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Manchester
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United Kingdom
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Norfolk
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United Kingdom
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Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Principia Biopharma, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04562766
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free number for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04562766