Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
NCT ID: NCT04562766
Last Updated: 2025-02-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
232 participants
INTERVENTIONAL
2020-12-14
2026-08-26
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Rilzabrutinib
Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period
Rilzabrutinib
400mg Caplet
Placebo
Patients receive matching placebo 400mg orally twice daily for up to 24 weeks
Placebo
400mg Caplet
Interventions
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Rilzabrutinib
400mg Caplet
Placebo
400mg Caplet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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
10 Years
ALL
No
Sponsors
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Principia Biopharma, a Sanofi Company
INDUSTRY
Responsible Party
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Locations
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University of Southern California_Investigational Site Number 84024
Los Angeles, California, United States
UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020
San Francisco, California, United States
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037
Torrance, California, United States
The Oncology Institute of Hope and Innovation_Investigational Site Number 84031
Whittier, California, United States
Children's Hospital Colorado_Investigational Site Number 84025
Aurora, Colorado, United States
IMMUNOe International Research Centers_Investigational Site Number 84028
Centennial, Colorado, United States
ASCLEPES Research Centers_Investigational Site Number 84023
Weeki Wachee, Florida, United States
Children's Healthcare of Atlanta_Investigational Site Number 84034
Atlanta, Georgia, United States
Rush University Medical Center_Investigational Site Number 84029
Chicago, Illinois, United States
University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033
Louisville, Kentucky, United States
Massachusetts General Hospital Site Number : 84038
Boston, Massachusetts, United States
Montefiore Medical Center_Investigational Site Number 84032
The Bronx, New York, United States
University Hospitals Cleveland Medical Center Site Number : 84036
Cleveland, Ohio, United States
Cleveland Clinic_Investigational Site Number 84026
Cleveland, Ohio, United States
The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027
Philadelphia, Pennsylvania, United States
University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035
Salt Lake City, Utah, United States
University of Washington Medical Centre Site Number : 84041
Seattle, Washington, United States
Investigational Site Number : 3206
Capital Federal, Buenos Aires, Argentina
Investigational Site Number : 3211
La Plata, Buenos Aires, Argentina
Investigational Site Number : 3205
Córdoba, Córdoba Province, Argentina
Investigational Site Number : 3209
Corrientes, , Argentina
Investigational Site Number : 3208
San Juan, , Argentina
Investigational Site Number : 3607
Kogarah, New South Wales, Australia
Investigational Site Number : 3608
Westmead, New South Wales, Australia
Investigational Site Number : 3611
Brisbane, Queensland, Australia
Investigational Site Number : 3609
Adelaide, South Australia, Australia
Investigational Site Number : 3606
Frankston, Victoria, Australia
Investigational Site Number : 3610
Perth, Western Australia, 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, Brazil
Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610
Cascavel, Paraná, Brazil
Hospital De Clinicas De Porto Alegre Site Number : 7605
Porto Alegre, Rio Grande do Sul, Brazil
Hospital Santa Marcelina Site Number : 7611
São Paulo, São Paulo, Brazil
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
Investigational Site Number : 12404
Edmonton, Alberta, Canada
Investigational Site Number : 12406
Toronto, Ontario, Canada
CHU Sainte-Justine_Investigational site number 12405
Montreal, Quebec, Canada
Investigational Site Number : 15201
La Serena, Coquimbo Region, Chile
Investigational Site Number : 15204
Santiago, Reg Metropolitana de Santiago, Chile
Investigational Site Number : 15202
Viña del Mar, Región de Valparaíso, Chile
Wuhan Union Hospital of Tongji Medical College of HUST - Investigational Site Number: 15601
Wuhan, Hubei, China
Shengjing Hospital of China Medical University - Investigational Site Number: 15603
Shenyang, Liaoning, China
Shaanxi Provincial People's Hospital - Investigational Site Number: 15607
Xi'an, Shaanxi, China
Qilu Hospital of Shandong University - Investigational Site Number: 15605
Jinan, Shandong, China
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences - Investigational Site Number: 15602
Tianjin, Tianjin Municipality, China
Second Affiliated Hospital of Kunming Medical University - Investigational Site Number: 15604
Kunming, Yunnan, China
Investigational Site Number : 15611
Hangzhou, , China
Investigational Site Number : 15608
Hefei, , China
Investigational Site Number : 15610
Nanchang, , China
Investigational Site Number : 15613
Tangshan, , China
Investigational Site Number : 15609
Wuxi, , China
Investigational Site Number : 15614
Zhenjiang, , China
Investigational Site Number : 25014
Angers, , France
Investigational Site Number : 25011
Créteil, , France
Investigational Site Number : 25010
Dijon, , France
Investigational Site Number : 25009
Nantes, , France
Investigational Site Number : 25008
Paris, , France
Investigational Site Number : 25012
Paris, , France
Investigational Site Number : 25007
Pessac, , France
Investigational Site Number : 27610
Berlin, , Germany
Investigational Site Number 27612
Düsseldorf, , Germany
Investigational Site Number : 27613
Frankfurt am Main, , Germany
Investigational Site Number : 27611
Recklinghausen, , Germany
Investigational Site Number : 34803
Budapest, , Hungary
Investigational Site Number : 34805
Debrecen, , Hungary
Investigational Site Number : 34801
Győr, , Hungary
Investigational Site Number : 34804
Nyíregyháza, , Hungary
Investigational Site Number : 34802
Székesfehérvár, , Hungary
Investigational Site Number : 37605
Haifa, , Israel
Investigational Site Number : 37606
Kfar Saba, , Israel
Investigational Site Number : 37607
Tel Aviv, , Israel
Investigational Site Number : 37608
Tel Litwinsky, , Israel
Investigational Site Number : 37609
Ẕerifin, , Israel
Investigational Site Number : 38014
Florence, Tuscany, Italy
Investigational Site Number : 38012
Bologna, , Italy
Investigational Site Number : 38015
Milan, , Italy
Investigational Site Number : 38013
Milan, , Italy
Investigational Site Number : 38010
Trieste, , Italy
Investigational Site Number : 38011
Vicenza, , Italy
Investigational Site Number : 39214
Tsuchiura-shi, Ibaraki, Japan
Investigational Site Number : 39205
Kanazawa, Ishikawa-ken, Japan
Investigational Site Number : 39207
Sagamihara-shi, Kanagawa, Japan
Investigational Site Number : 39202
Suita-shi, Osaka, Japan
Investigational Site Number : 39201
Iruma-gun, Saitama, Japan
Investigational Site Number : 39208
Bunkyo-ku, Tokyo, Japan
Investigational Site Number : 39210
Meguro-ku, Tokyo, Japan
Investigational Site Number : 39204
Setagaya City, Tokyo, Japan
Investigational Site Number : 39209
Sumida-ku, Tokyo, Japan
Investigational Site Number : 39212
Chiba, , Japan
Investigational Site Number : 39203
Hiroshima, , Japan
Investigational Site Number : 39206
Saitama-shi, , Japan
Investigational Site Number : 48401
Monterrey, Nuevo León, Mexico
Investigational Site Number : 48402
Chihuahua City, , Mexico
Investigational Site Number : 48405
Delegacion Benito Juarez, , Mexico
Investigational Site Number : 48404
Durango, , Mexico
Investigational Site Number : 48406
Mexico City, , Mexico
Investigational Site Number : 48403
Zapopan, , Mexico
Erasmus MC_Investigational Site Number 52801
Rotterdam, , Netherlands
Investigational Site Number : 57802
Bergen, , Norway
Investigational Site Number : 57801
Grålum, , Norway
Investigational Site Number : 61617
Piła, Greater Poland Voivodeship, Poland
Investigational Site Number : 61609
Słupsk, Pomeranian Voivodeship, Poland
Investigational Site Number : 61615
Gdynia, , Poland
Investigational Site Number : 61614
Poznan, , Poland
Investigational Site Number : 61612
Warsaw, , Poland
Investigational Site Number : 61613
Wroclaw, , Poland
Investigational Site Number : 64307
Moscow, , Russia
Investigational Site Number : 64305
Moscow, , Russia
Investigational Site Number : 64304
Novosibirsk, , Russia
Investigational Site Number : 64301
Pyatigorsk, , Russia
Investigational Site Number : 64302
Saint Petersburg, , Russia
Investigational Site Number : 64306
Samara, , Russia
Investigational Site Number : 64303
Tula, , Russia
Investigational Site Number : 70201
Singapore, , Singapore
Investigational Site Number : 70202
Singapore, , Singapore
Investigational Site Number : 70203
Singapore, , Singapore
Investigational Site Number : 41004
Busan, Busan, South Korea
Investigational Site Number : 41001
Suwon, Gyeonggi-do, South Korea
Investigational Site Number : 41003
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 41005
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 41006
Seoul, Seoul-teukbyeolsi, South Korea
Investigational Site Number : 72408
Málaga, Málaga, Spain
Investigational Site Number : 72407
Valencia, Valenciana, Comunidad, Spain
Investigational Site Number : 72412
Barcelona, , Spain
Investigational Site Number : 72414
Barcelona, , Spain
Investigational Site Number : 72409
Barcelona, , Spain
Investigational Site Number : 72416
Burgos, , Spain
Investigational Site Number : 72410
Madrid, , Spain
Investigational Site Number : 72411
Murcia, , Spain
Investigational Site Number : 72413
Seville, , Spain
Investigational Site Number : 76405
Bangkok, , Thailand
Investigational Site Number : 76404
Bangkok, , Thailand
Investigational Site Number : 76402
Chiang Mai, , Thailand
Investigational Site Number : 76401
Khon Kaen, , Thailand
Investigational Site Number : 76403
Songkhla, , Thailand
Investigational Site Number 79208
Ankara, , Turkey (Türkiye)
Investigational Site Number 79210
Istanbul, , Turkey (Türkiye)
Investigational Site Number 79206
Izmir, , Turkey (Türkiye)
Investigational Site Number 79209
Kayseri, , Turkey (Türkiye)
Investigational Site Number : 80408
Dnipropetrovsk, , Ukraine
Investigational Site Number : 80409
Kryvyi Rih, , Ukraine
Investigational Site Number : 80410
Kyiv, , Ukraine
Investigational Site Number : 82607
London, London, City of, United Kingdom
Investigational Site Number : 82604
Harrow, , United Kingdom
Investigational Site Number : 82606
London, , United Kingdom
Investigational Site Number : 82609
London, , United Kingdom
Investigational Site Number : 82603
Manchester, , United Kingdom
Investigational Site Number : 82605
Norfolk, , United Kingdom
Investigational Site Number : 82608
Southampton, , United Kingdom
Countries
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References
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Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Homenda W, Galindo LEM, Basquiera AL, Tan CW, Saydam G, Hutter-Kronke ML, Chai-Adisaksopha C, Gomez-Almaguer D, Tran H, Shin HJ, Dantas da Cunha Junior A, Lazar Z, Izquierdo CP, Kirgner I, Lucchini E, Kuzmina G, Fillitz M, Audia S, Taparia M, Cordoba M, Diab R, Yao M, Gouia I, Lee M, Daak A. Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study. Blood. 2025 Jun 12;145(24):2914-2926. doi: 10.1182/blood.2024027336.
Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available.
Kuter DJ, Bussel JB, Ghanima W, Cooper N, Gernsheimer T, Lambert MP, Liebman HA, Tarantino MD, Lee M, Guo H, Daak A. Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. Ther Adv Hematol. 2023 Oct 18;14:20406207231205431. doi: 10.1177/20406207231205431. eCollection 2023.
Other Identifiers
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PRN1008-018
Identifier Type: OTHER
Identifier Source: secondary_id
2023-509401-71
Identifier Type: REGISTRY
Identifier Source: secondary_id
EFC17093
Identifier Type: -
Identifier Source: org_study_id
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