Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA 3)

April 10, 2024 updated by: Principia Biopharma, a Sanofi Company

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)

This is 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 will receive rilzabrutinib or placebo 400mg twice daily.

For each patient, 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

194

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Trial Transparency email recommended (Toll free number for US & Canada)
  • Phone Number: option 6 800-633-1610
  • Email: Contact-US@sanofi.com

Study Locations

  • Argentina
      • Corrientes, Argentina, W3410 FND
        • Active, not recruiting
        • Investigational Site Number : 3209
      • San Juan, Argentina, 5400
        • Active, not recruiting
        • Investigational Site Number : 3208
    • Buenos Aires
      • Capital Federal, Buenos Aires, Argentina, C1280AEB
        • Active, not recruiting
        • Investigational Site Number : 3206
      • La Plata, Buenos Aires, Argentina, B1900
        • Completed
        • Investigational Site Number : 3211
    • Córdoba
      • Cordoba, Córdoba, Argentina, X5003DCE
        • Recruiting
        • Investigational Site Number : 3205
  • Australia
    • New South Wales
      • Kogarah, New South Wales, Australia, 2217
        • Active, not recruiting
        • Investigational Site Number : 3607
      • Westmead, New South Wales, Australia, 2145
        • Active, not recruiting
        • Investigational Site Number : 3608
    • Queensland
      • Brisbane, Queensland, Australia, 4101
        • Recruiting
        • Investigational Site Number : 3611
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Active, not recruiting
        • Investigational Site Number : 3609
    • Victoria
      • Frankston, Victoria, Australia, 3199
        • Active, not recruiting
        • Investigational Site Number : 3606
    • Western Australia
      • Perth, Western Australia, Australia, 6000
        • Active, not recruiting
        • Investigational Site Number : 3610
  • Austria
      • Graz, Austria, 8036
        • Active, not recruiting
        • Investigational Site Number : 4005
      • Leoben, Austria, 8700
        • Active, not recruiting
        • Investigational Site Number : 4004
      • Linz, Austria, A4020
        • Active, not recruiting
        • Investigational Site Number : 4001
      • Steyr, Austria, 4400
        • Completed
        • Investigational Site Number : 4003
      • Wien, Austria, 1140
        • Active, not recruiting
        • Investigational Site Number : 4002
  • Brazil
      • Belem Do Para, Brazil, 66053-000
        • Active, not recruiting
        • CEMEC Oncologica do Brasil Site Number : 7606
      • Rio de Janeiro, Brazil, 20211030
        • Active, not recruiting
        • HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 7609
      • São Paulo, Brazil, 04039-004
        • Completed
        • Hospital do Servidor Publico Estadual de Sao Paulo Site Number : 7607
    • Bahia
      • Salvador, Bahia, Brazil, 41253190
        • Recruiting
        • Hospital Sao Rafael Instituto D'Or da Bahia Site Number : 7608
    • Paraná
      • Cascavel, Paraná, Brazil, 85806-300
        • Recruiting
        • Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610
    • Rio Grande Do Sul
      • Porto Alegre, Rio Grande Do Sul, Brazil, 90035 003
        • Active, not recruiting
        • Hospital De Clinicas De Porto Alegre Site Number : 7605
    • São Paulo
      • Sao Paulo, São Paulo, Brazil, 08270-070
        • Recruiting
        • Hospital Santa Marcelina Site Number : 7611
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2P4
        • Active, not recruiting
        • Investigational Site Number : 12404
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Recruiting
        • Investigational Site Number : 12406
    • Quebec
  • Chile
    • Coquimbo
      • La Serena, Coquimbo, Chile, 1720430
        • Active, not recruiting
        • Investigational Site Number : 15201
    • Reg Metropolitana De Santiago
      • Santiago, Reg Metropolitana De Santiago, Chile, 7500653
        • Active, not recruiting
        • Investigational Site Number : 15204
    • Valparaíso
      • Vina del Mar, Valparaíso, Chile, 322000
        • Active, not recruiting
        • Investigational Site Number : 15202
  • China
      • Hangzhou, China, 310018
        • Recruiting
        • Investigational Site Number : 15611
      • Hefei, China
        • Recruiting
        • Investigational Site Number : 15608
      • Nanchang, China, 330006
        • Recruiting
        • Investigational Site Number : 15610
      • Tangshan, China, 63000
        • Recruiting
        • Investigational Site Number : 15613
      • Wuxi, China, 214023
        • Active, not recruiting
        • Investigational Site Number : 15609
      • ZhenJiang, China, 212001
        • Recruiting
        • Investigational Site Number : 15614
    • Hubei
      • Wuhan, Hubei, China, 430022
        • Recruiting
        • Wuhan Union Hospital of Tongji Medical College of HUST - Investigational Site Number: 15601
        • Contact:
        • Principal Investigator:
          • Yu Hu
    • Liaoning
      • Shenyang, Liaoning, China, 110022
        • Recruiting
        • Shengjing Hospital of China Medical University - Investigational Site Number: 15603
        • Contact:
        • Principal Investigator:
          • Zhuogang Liu
    • Shaanxi
      • Xi'an, Shaanxi, China, 710068
        • Recruiting
        • Shaanxi Provincial People's Hospital - Investigational Site Number: 15607
        • Contact:
        • Principal Investigator:
          • Yi Wang
    • Shandong
      • Jinan, Shandong, China, 250012
        • Active, not recruiting
        • Qilu Hospital of Shandong University - Investigational Site Number: 15605
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Recruiting
        • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences - Investigational Site Number: 15602
        • Contact:
        • Principal Investigator:
          • Lei Zhang
    • Yunnan
      • Kunming, Yunnan, China, 650101
        • Recruiting
        • Second Affiliated Hospital of Kunming Medical University - Investigational Site Number: 15604
        • Principal Investigator:
          • Zeping Zhou
        • Contact:
  • France
      • Angers, France, 49933
        • Active, not recruiting
        • Investigational Site Number : 25014
      • Creteil, France, 94000
        • Active, not recruiting
        • Investigational Site Number : 25011
      • Dijon cedex, France, 21079
        • Active, not recruiting
        • Investigational Site Number : 25010
      • Nantes Cedex 1, France, 44093
        • Active, not recruiting
        • Investigational Site Number : 25009
      • Paris, France, 75019
        • Recruiting
        • Investigational Site Number : 25012
      • Paris, France, 75012
        • Active, not recruiting
        • Investigational Site Number : 25008
      • Pessac, France, 33600
        • Active, not recruiting
        • Investigational Site Number : 25007
  • Germany
      • Berlin, Germany, 10117
        • Active, not recruiting
        • Investigational Site Number : 27610
      • Düsseldorf, Germany, 40497
        • Active, not recruiting
        • Investigational Site Number 27612
      • Frankfurt am Main, Germany, 60596
        • Active, not recruiting
        • Investigational Site Number : 27613
      • Recklinghausen, Germany, 45659
        • Active, not recruiting
        • Investigational Site Number : 27611
  • Hungary
      • Budapest, Hungary, 1083
        • Active, not recruiting
        • Investigational Site Number : 34803
      • Debrecen, Hungary, 4032
        • Completed
        • Investigational Site Number : 34805
      • Gyor, Hungary, 9024
        • Active, not recruiting
        • Investigational Site Number : 34801
      • Nyíregyháza, Hungary, 4405
        • Active, not recruiting
        • Investigational Site Number : 34804
      • Szekesfehervar, Hungary, 8000
        • Recruiting
        • Investigational Site Number : 34802
  • Israel
      • Haifa, Israel, 3109601
        • Active, not recruiting
        • Investigational Site Number : 37605
      • Kfar Saba, Israel, 4428164
        • Active, not recruiting
        • Investigational Site Number : 37606
      • Tel Aviv, Israel, 6423906
        • Active, not recruiting
        • Investigational Site Number : 37607
      • Tel Hashomer, Israel, 52621
        • Recruiting
        • Investigational Site Number : 37608
      • Zerifin, Israel, 70300
        • Active, not recruiting
        • Investigational Site Number : 37609
  • Italy
      • Bologna, Italy, 40138
        • Active, not recruiting
        • Investigational Site Number : 38012
      • Milano, Italy, 20123
        • Active, not recruiting
        • Investigational Site Number : 38015
      • Milano, Italy, 20142
        • Active, not recruiting
        • Investigational Site Number : 38013
      • Trieste, Italy, 34125
        • Active, not recruiting
        • Investigational Site Number : 38010
      • Vicenza, Italy, 36100
        • Active, not recruiting
        • Investigational Site Number : 38011
    • Toscana
      • Firenze, Toscana, Italy, 50134
        • Active, not recruiting
        • Investigational Site Number : 38014
  • Japan
      • Chiba, Japan, 60-0852
        • Active, not recruiting
        • Investigational Site Number : 39212
      • Hiroshima City, Japan, 730-8619
        • Active, not recruiting
        • Investigational Site Number : 39203
      • Saitama-shi, Japan, 330-8777
        • Recruiting
        • Investigational Site Number : 39206
    • Ibaraki
      • Tsuchiura-shi, Ibaraki, Japan
        • Active, not recruiting
        • Investigational Site Number : 39214
    • Ishikawa
      • Kanazawa-shi, Ishikawa, Japan, 920-8530
        • Active, not recruiting
        • Investigational Site Number : 39205
    • Kanagawa
      • Sagamihara-shi, Kanagawa, Japan, 252-0375
        • Active, not recruiting
        • Investigational Site Number : 39207
    • Osaka
      • Suita-shi, Osaka, Japan, 565-0871
        • Completed
        • Investigational Site Number : 39202
    • Saitama
      • Iruma-gun, Saitama, Japan, 350-0495
        • Active, not recruiting
        • Investigational Site Number : 39201
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 113-8655
        • Recruiting
        • Investigational Site Number : 39208
      • Meguro-ku, Tokyo, Japan, 152-8902
        • Completed
        • Investigational Site Number : 39210
      • Setagaya, Tokyo, Japan, 157-0074
        • Recruiting
        • Investigational Site Number : 39204
      • Sumida-ku, Tokyo, Japan, 130-8575
        • Active, not recruiting
        • Investigational Site Number : 39209
  • Korea, Republic of
    • Busan-gwangyeoksi
      • Busan, Busan-gwangyeoksi, Korea, Republic of, 49241
        • Recruiting
        • Investigational Site Number : 41004
    • Gyeonggi-do
      • Suwon, Gyeonggi-do, Korea, Republic of, 16499
        • Active, not recruiting
        • Investigational Site Number : 41001
    • Seoul-teukbyeolsi
      • Seoul, Seoul-teukbyeolsi, Korea, Republic of, 03080
        • Recruiting
        • Investigational Site Number : 41005
      • Seoul, Seoul-teukbyeolsi, Korea, Republic of, 03722
        • Recruiting
        • Investigational Site Number : 41006
      • Seoul, Seoul-teukbyeolsi, Korea, Republic of, 02841
        • Active, not recruiting
        • Investigational Site Number : 41003
  • Mexico
      • Chihuahua, Mexico, 31200
        • Active, not recruiting
        • Investigational Site Number : 48402
      • Ciudad de México, Mexico, 06760
        • Recruiting
        • Investigational Site Number : 48406
      • Delegacion Benito Juarez, Mexico, 03720
        • Active, not recruiting
        • Investigational Site Number : 48405
      • Durango, Mexico, 34000
        • Recruiting
        • Investigational Site Number : 48404
      • Zapopan, Mexico, 45030
        • Active, not recruiting
        • Investigational Site Number : 48403
    • Nuevo León
      • Monterrey, Nuevo León, Mexico, 64460
        • Active, not recruiting
        • Investigational Site Number : 48401
  • Netherlands
      • Rotterdam, Netherlands, 3015 GD
        • Active, not recruiting
        • Erasmus MC_Investigational Site Number 52801
  • Norway
      • Bergen, Norway, 5021
        • Active, not recruiting
        • Investigational Site Number : 57802
      • Gralum, Norway, 1714
        • Active, not recruiting
        • Investigational Site Number : 57801
  • Poland
      • Gdynia, Poland, 81-519
        • Active, not recruiting
        • Investigational Site Number : 61615
      • Poznan, Poland, 61 696
        • Active, not recruiting
        • Investigational Site Number : 61614
      • Warszawa, Poland, 02-776
        • Active, not recruiting
        • Investigational Site Number : 61612
      • Wroclaw, Poland, 50-556
        • Active, not recruiting
        • Investigational Site Number : 61613
    • Pomorskie
      • Slupsk, Pomorskie, Poland, 76-200
        • Active, not recruiting
        • Investigational Site Number : 61609
    • Wielkopolskie
      • Pila, Wielkopolskie, Poland, 64-920
        • Completed
        • Investigational Site Number : 61617
  • Russian Federation
      • Moscow, Russian Federation, 119049
        • Recruiting
        • Investigational Site Number : 64307
      • Moscow, Russian Federation, 125167
        • Completed
        • Investigational Site Number : 64305
      • Novosibirsk, Russian Federation, 630090
        • Recruiting
        • Investigational Site Number : 64304
      • Pyatigorsk, Russian Federation, 357502
        • Recruiting
        • Investigational Site Number : 64301
      • Samara, Russian Federation, 443099
        • Completed
        • Investigational Site Number : 64306
      • St. Petersburg, Russian Federation, 191024
        • Active, not recruiting
        • Investigational Site Number : 64302
      • Tula, Russian Federation, 300053
        • Completed
        • Investigational Site Number : 64303
  • Singapore
      • Singapore, Singapore, 119228
        • Recruiting
        • Investigational Site Number : 70201
      • Singapore, Singapore, 169608
        • Active, not recruiting
        • Investigational Site Number : 70202
      • Singapore, Singapore, 308433
        • Active, not recruiting
        • Investigational Site Number : 70203
  • Spain
      • Barcelona, Spain, 08035
        • Active, not recruiting
        • Investigational Site Number : 72412
      • Barcelona, Spain, 08035
        • Active, not recruiting
        • Investigational Site Number : 72414
      • Barcelona, Spain, 08041
        • Active, not recruiting
        • Investigational Site Number : 72409
      • Burgos, Spain
        • Recruiting
        • Investigational Site Number : 72416
      • Madrid, Spain, 28007
        • Recruiting
        • Investigational Site Number : 72410
      • Murcia, Spain, 30008
        • Active, not recruiting
        • Investigational Site Number : 72411
      • Sevilla, Spain, 41013
        • Recruiting
        • Investigational Site Number : 72413
    • Málaga
      • Malaga, Málaga, Spain, 29010
        • Recruiting
        • Investigational Site Number : 72408
    • Valenciana, Comunidad
      • Valencia / Valencia, Valenciana, Comunidad, Spain, 46010
        • Active, not recruiting
        • Investigational Site Number : 72407
  • Thailand
      • Bangkok, Thailand, 10330
        • Active, not recruiting
        • Investigational Site Number : 76405
      • Bangkok, Thailand, 10400
        • Active, not recruiting
        • Investigational Site Number : 76404
      • Chiangmai, Thailand, 50200
        • Active, not recruiting
        • Investigational Site Number : 76402
      • Khon Kaen, Thailand, 40002
        • Recruiting
        • Investigational Site Number : 76401
      • Songkla, Thailand, 90110
        • Recruiting
        • Investigational Site Number : 76403
  • Turkey
      • Ankara, Turkey, 06620
        • Active, not recruiting
        • Investigational Site Number 79208
      • Istanbul, Turkey, 34093
        • Active, not recruiting
        • Investigational Site Number 79210
      • Izmir, Turkey, 35100
        • Active, not recruiting
        • Investigational Site Number 79206
      • Kayseri, Turkey, 38039
        • Active, not recruiting
        • Investigational Site Number 79209
  • Ukraine
      • Dnipropetrovsk, Ukraine, 49102
        • Active, not recruiting
        • Investigational Site Number : 80408
      • Kryvyi Rih City, Ukraine, 50025
        • Active, not recruiting
        • Investigational Site Number : 80409
      • Kyiv, Ukraine, 03143
        • Active, not recruiting
        • Investigational Site Number : 80410
  • United Kingdom
      • Harrow, United Kingdom, HA1 3UJ
        • Active, not recruiting
        • Investigational Site Number : 82604
      • London, United Kingdom, W2 1NY
        • Recruiting
        • Investigational Site Number : 82609
      • London, United Kingdom, SE5 9PJ
        • Active, not recruiting
        • Investigational Site Number : 82606
      • Manchester, United Kingdom, M13 9WL
        • Recruiting
        • Investigational Site Number : 82603
      • Norfolk, United Kingdom, NR31 6LA
        • Completed
        • Investigational Site Number : 82605
      • Southampton, United Kingdom, SO16 6YD
        • Recruiting
        • Investigational Site Number : 82608
    • London, City Of
      • London, London, City Of, United Kingdom, W12 0HS
        • Active, not recruiting
        • Investigational Site Number : 82607
  • United States
    • California
      • Los Angeles, California, United States, 90033
        • Active, not recruiting
        • University of Southern California_Investigational Site Number 84024
      • San Francisco, California, United States, 94158
        • Recruiting
        • UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020
        • Contact:
        • Principal Investigator:
          • Kristin Shimano
      • Torrance, California, United States, 90502
        • Active, not recruiting
        • Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037
      • Whittier, California, United States, 90602
        • Active, not recruiting
        • The Oncology Institute of Hope and Innovation_Investigational Site Number 84031
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado_Investigational Site Number 84025
        • Contact:
        • Principal Investigator:
          • Taizo Nakano
      • Centennial, Colorado, United States, 80112
        • Recruiting
        • IMMUNOe International Research Centers_Investigational Site Number 84028
        • Contact:
        • Principal Investigator:
          • Isaac Melamed, MD
    • Florida
      • Weeki Wachee, Florida, United States, 34607
        • Active, not recruiting
        • ASCLEPES Research Centers_Investigational Site Number 84023
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Children's Healthcare of Atlanta_Investigational Site Number 84034
        • Contact:
        • Principal Investigator:
          • Carolyn Bennett
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Active, not recruiting
        • Rush University Medical Center_Investigational Site Number 84029
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Completed
        • University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Active, not recruiting
        • Massachusetts General Hospital Site Number : 84038
    • New York
      • Bronx, New York, United States, 10461
        • Recruiting
        • Montefiore Medical Center_Investigational Site Number 84032
        • Contact:
        • Principal Investigator:
          • Irina Murakhovskaya
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • University Hospitals Cleveland Medical Center Site Number : 84036
      • Cleveland, Ohio, United States, 44195
        • Active, not recruiting
        • Cleveland Clinic_Investigational Site Number 84026
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027
        • Contact:
        • Principal Investigator:
          • Michele Lambert
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Active, not recruiting
        • University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035
    • Washington
      • Seattle, Washington, United States, 98195
        • Active, not recruiting
        • University of Washington Medical Centre Site Number : 84041

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Patients 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. 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])
  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. 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); 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Rilzabrutinib
Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period
Drug: Rilzabrutinib
400mg Caplet
Other Names:
  • PRN1008
Placebo Comparator: Placebo
Patients receive matching placebo 400mg orally twice daily for up to 24 weeks
Drug: Placebo
400mg Caplet
Other Names:
  • PRN1008 Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Durable platelet response during the last 6 weeks of the 24-week blinded treatment period (not for EU and UK)
Time Frame: 24 weeks
Durable platelet response is defined as a proportion of participants able to achieve platelet counts at or above 50,000/μL 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 are at or above 50,000/μL.
24 weeks
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
Time Frame: 24 weeks
24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Time Frame: 24 weeks
24 weeks
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
Time Frame: 24 weeks
24 weeks
Time to first platelet count of ≥50,000/μL OR between ≥30,000/μL and <50,000/μL and doubled from baseline
Time Frame: 24 weeks
24 weeks
Proportion of patients requiring rescue therapy during the 24-week blinded treatment period
Time Frame: 24 weeks
24 weeks
Change from baseline on Item 10 of the ITP-Patient Assessment Questionnaire in adult patients (≥18 years) at Week 13
Time Frame: From baseline to Week 13
From baseline to Week 13
for EU and UK: Change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25
Time Frame: At Week 25
At Week 25
Proportion of participants who able to achieve stable platelet response, within a period of 24 weeks following initial achievement of the platelet response
Time Frame: 24 weeks
Stable platelet response is defined as no 2 scheduled visits, at least 4 weeks apart, with a platelet count less than 50,000/µL, without an intervening visit with a platelet count ≥50,000/µL. Initial platelet response defined as platelet count ≥50,000/μL within 12 weeks of initiation of treatment with rilzabrutinib during the study.
24 weeks
Frequency and severity of Treatment Emergent Adverse Events
Time Frame: 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Including physical examination, ECG, clinical laboratory test results, vital signs and laboratory tests (serum chemistry, hematology, except for platelet counts included in the primary efficacy endpoint)
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Frequency and severity of bleeding TEAEs
Time Frame: 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Plasma concentrations of rilzabrutinib
Time Frame: Until 52 weeks
Until 52 weeks
Change from baseline on the Symptoms, Bother and Activity domains of the ITP Patient Assessment Questionnaire (ITP-PAQ) in adult patients (≥18 years)
Time Frame: 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
The ITP Patient Assessment Questionnaire™ (ITP-PAQ™) is a disease-specific instrument that was designed to measure the Quality of Life (QoL) of adult patients with immune thrombocytopenia. The items employ a 4-week recall with responses recorded on 4-, 5- or 7-point Likert scales. All item scores are transformed to a 0 to 100 continuum where higher scores represent better QoL and are weighted equally to derive the scale scores.
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
Change from baseline in disease-specific QoL as measured by the Kids' ITP Tools (ITP-KIT) score in pediatric participants
Time Frame: 52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose
The ITP-KIT include a battery of three disease-specific instruments, a child self-report form designed to be completed by children ≥7 years, a parent proxy report form for children <7 and a parent impact form. Respondents record their disease experience based on a 1-week recall. The instrument yields a total score which is the summation of the items converted to a 0 to 100 score with higher scores indicating better disease-specific QoL.
52 weeks of treatment, 12 months of long term extension and 4 weeks of follow up post last dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Principia Biopharma, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 14, 2020

Primary Completion (Estimated)

June 5, 2025

Study Completion (Estimated)

November 20, 2026

Study Registration Dates

First Submitted

September 18, 2020

First Submitted That Met QC Criteria

September 18, 2020

First Posted (Actual)

September 24, 2020

Study Record Updates

Last Update Posted (Actual)

April 11, 2024

Last Update Submitted That Met QC Criteria

April 10, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EFC17093
  • PRN1008-018 (Other Identifier: Principia Biopharma)
  • 2023-509401-71 (Registry Identifier: CTIS)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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