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

NCT04562766 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 3 other identifiers: EFC17093PRN1008-0182023-509401-71
• Study Type: interventional
• Target: 232
• Locations: 25 countries
• Sites: 149

Brief 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.

Conditions

Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (2)

• DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom

  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.

  Up to 24 weeks

• DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom

  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.

  Up to 24 weeks

Secondary Outcomes (13)

• DB Period: Number of Weeks With Platelet Count >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy

  Up to 24 weeks

• DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy

  Up to 24 weeks

• DB Period: Time to First Platelet Count of >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and Doubled From Baseline

  Up to 24 weeks

• DB Period: Percentage of Participants Who Required Rescue Therapy

  Up to 24 weeks

• DB Period: Change From Baseline on Item 10 (Physical Fatigue) of the Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) in Adult Participants at Week 13

  Baseline (Day 1) and Week 13

Study Arms (2)

Rilzabrutinib

EXPERIMENTAL

Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period

Drug: Rilzabrutinib

Placebo

PLACEBO COMPARATOR

Patients receive matching placebo 400mg orally twice daily for up to 24 weeks

Drug: Placebo

Interventions

Rilzabrutinib DRUG

400mg Caplet

Also known as: PRN1008

Rilzabrutinib

Placebo DRUG

400mg Caplet

Also known as: PRN1008 Placebo

Placebo

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• \- Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator.
• 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 \[Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants\])
• 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

You may not qualify if:

• 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
• \- Patients treated with rituximab will have normal B-cell counts prior to enrollment
• 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
• History of solid organ transplant
• Myelodysplastic syndrome
• Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
• Planned surgery in the time frame of the dosing period

Sponsors & Collaborators

• Principia Biopharma, a Sanofi Company: lead

Study Sites (155)

University of Southern California_Investigational Site Number 84024

Los Angeles, California, 90033, United States

Location

UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020

San Francisco, California, 94158, United States

Location

Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037

Torrance, California, 90502, United States

Location

The Oncology Institute of Hope and Innovation_Investigational Site Number 84031

Whittier, California, 90602, United States

Location

Children's Hospital Colorado_Investigational Site Number 84025

Aurora, Colorado, 80045, United States

Location

IMMUNOe International Research Centers_Investigational Site Number 84028

Centennial, Colorado, 80112, United States

Location

ASCLEPES Research Centers_Investigational Site Number 84023

Weeki Wachee, Florida, 34607, United States

Location

Children's Healthcare of Atlanta_Investigational Site Number 84034

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center_Investigational Site Number 84029

Chicago, Illinois, 60612, United States

Location

University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033

Louisville, Kentucky, 40202, United States

Location

Massachusetts General Hospital Site Number : 84038

Boston, Massachusetts, 02114, United States

Location

Montefiore Medical Center_Investigational Site Number 84032

The Bronx, New York, 10461, United States

Location

University Hospitals Cleveland Medical Center Site Number : 84036

Cleveland, Ohio, 44106, United States

Location

Cleveland Clinic_Investigational Site Number 84026

Cleveland, Ohio, 44195, United States

Location

The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027

Philadelphia, Pennsylvania, 19104, United States

Location

University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035

Salt Lake City, Utah, 84112, United States

Location

University of Washington Medical Centre Site Number : 84041

Seattle, Washington, 98195, United States

Location

Investigational Site Number : 3206

Capital Federal, Buenos Aires, C1280AEB, Argentina

Location

Investigational Site Number : 3211

La Plata, Buenos Aires, B1900, Argentina

Location

Investigational Site Number : 3205

Córdoba, Córdoba Province, X5003DCE, Argentina

Location

Investigational Site Number : 3209

Corrientes, W3410 FND, Argentina

Location

Investigational Site Number : 3208

San Juan, 5400, Argentina

Location

Investigational Site Number : 3607

Kogarah, New South Wales, 2217, Australia

Location

Investigational Site Number : 3608

Westmead, New South Wales, 2145, Australia

Location

Investigational Site Number : 3611

Brisbane, Queensland, 4101, Australia

Location

Investigational Site Number : 3609

Adelaide, South Australia, 5000, Australia

Location

Investigational Site Number : 3606

Frankston, Victoria, 3199, Australia

Location

Investigational Site Number : 3610

Perth, Western Australia, 6000, Australia

Location

Investigational Site Number : 4005

Graz, 8036, Austria

Location

Investigational Site Number : 4004

Leoben, 8700, Austria

Location

Investigational Site Number : 4001

Linz, A4020, Austria

Location

Investigational Site Number : 4003

Steyr, 4400, Austria

Location

Investigational Site Number : 4002

Vienna, 1140, Austria

Location

Hospital Sao Rafael Instituto D'Or da Bahia Site Number : 7608

Salvador, Estado de Bahia, 41253190, Brazil

Location

Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610

Cascavel, Paraná, 85806-300, Brazil

Location

Hospital De Clinicas De Porto Alegre Site Number : 7605

Porto Alegre, Rio Grande do Sul, 90035 003, Brazil

Location

Hospital Santa Marcelina Site Number : 7611

São Paulo, São Paulo, 08270-070, Brazil

Location

CEMEC Oncologica do Brasil Site Number : 7606

Belém, 66053-000, Brazil

Location

HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 7609

Rio de Janeiro, 20211030, Brazil

Location

Hospital do Servidor Publico Estadual de Sao Paulo Site Number : 7607

São Paulo, 04039-004, Brazil

Location

Investigational Site Number : 12404

Edmonton, Alberta, T6G 2P4, Canada

Location

Investigational Site Number : 12406

Toronto, Ontario, M5G 1X8, Canada

Location

CHU Sainte-Justine_Investigational site number 12405

Montreal, Quebec, H3T 1C5, Canada

Location

Investigational Site Number : 15201

La Serena, Coquimbo Region, 1720430, Chile

Location

Investigational Site Number : 15204

Santiago, Reg Metropolitana de Santiago, 7500653, Chile

Location

Investigational Site Number : 15202

Viña del Mar, Región de Valparaíso, 322000, Chile

Location

Wuhan Union Hospital of Tongji Medical College of HUST - Investigational Site Number: 15601

Wuhan, Hubei, 430022, China

Location

Shengjing Hospital of China Medical University - Investigational Site Number: 15603

Shenyang, Liaoning, 110022, China

Location

Shaanxi Provincial People's Hospital - Investigational Site Number: 15607

Xi'an, Shaanxi, 710068, China

Location

Qilu Hospital of Shandong University - Investigational Site Number: 15605

Jinan, Shandong, 250012, China

Location

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences - Investigational Site Number: 15602

Tianjin, Tianjin Municipality, 300020, China

Location

Second Affiliated Hospital of Kunming Medical University - Investigational Site Number: 15604

Kunming, Yunnan, 650101, China

Location

Investigational Site Number : 15611

Hangzhou, 310018, China

Location

Investigational Site Number : 15608

Hefei, China

Location

Investigational Site Number : 15610

Nanchang, 330006, China

Location

Investigational Site Number : 15613

Tangshan, 63000, China

Location

Investigational Site Number : 15609

Wuxi, 214023, China

Location

Investigational Site Number : 15614

Zhenjiang, 212001, China

Location

Investigational Site Number : 25014

Angers, 49933, France

Location

Investigational Site Number : 25011

Créteil, 94000, France

Location

Investigational Site Number : 25010

Dijon, 21079, France

Location

Investigational Site Number : 25009

Nantes, 44093, France

Location

Investigational Site Number : 25008

Paris, 75012, France

Location

Investigational Site Number : 25012

Paris, 75019, France

Location

Investigational Site Number : 25007

Pessac, 33600, France

Location

Investigational Site Number : 27610

Berlin, 10117, Germany

Location

Investigational Site Number 27612

Düsseldorf, 40497, Germany

Location

Investigational Site Number : 27613

Frankfurt am Main, 60596, Germany

Location

Investigational Site Number : 27611

Recklinghausen, 45659, Germany

Location

Investigational Site Number : 34803

Budapest, 1083, Hungary

Location

Investigational Site Number : 34805

Debrecen, 4032, Hungary

Location

Investigational Site Number : 34801

Győr, 9024, Hungary

Location

Investigational Site Number : 34804

Nyíregyháza, 4405, Hungary

Location

Investigational Site Number : 34802

Székesfehérvár, 8000, Hungary

Location

Investigational Site Number : 37605

Haifa, 3109601, Israel

Location

Investigational Site Number : 37606

Kfar Saba, 4428164, Israel

Location

Investigational Site Number : 37607

Tel Aviv, 6423906, Israel

Location

Investigational Site Number : 37608

Tel Litwinsky, 52621, Israel

Location

Investigational Site Number : 37609

Ẕerifin, 70300, Israel

Location

Investigational Site Number : 38014

Florence, Tuscany, 50134, Italy

Location

Investigational Site Number : 38012

Bologna, 40138, Italy

Location

Investigational Site Number : 38015

Milan, 20123, Italy

Location

Investigational Site Number : 38013

Milan, 20142, Italy

Location

Investigational Site Number : 38010

Trieste, 34125, Italy

Location

Investigational Site Number : 38011

Vicenza, 36100, Italy

Location

Investigational Site Number : 39214

Tsuchiura-shi, Ibaraki, Japan

Location

Investigational Site Number : 39205

Kanazawa, Ishikawa-ken, 920-8530, Japan

Location

Investigational Site Number : 39207

Sagamihara-shi, Kanagawa, 252-0375, Japan

Location

Investigational Site Number : 39202

Suita-shi, Osaka, 565-0871, Japan

Location

Investigational Site Number : 39201

Iruma-gun, Saitama, 350-0495, Japan

Location

Investigational Site Number : 39208

Bunkyo-ku, Tokyo, 113-8655, Japan

Location

Investigational Site Number : 39210

Meguro-ku, Tokyo, 152-8902, Japan

Location

Investigational Site Number : 39204

Setagaya City, Tokyo, 157-0074, Japan

Location

Investigational Site Number : 39209

Sumida-ku, Tokyo, 130-8575, Japan

Location

Investigational Site Number : 39212

Chiba, 60-0852, Japan

Location

Investigational Site Number : 39203

Hiroshima, 730-8619, Japan

Location

Investigational Site Number : 39206

Saitama-shi, 330-8777, Japan

Location

Investigational Site Number : 48401

Monterrey, Nuevo León, 64460, Mexico

Location

Investigational Site Number : 48402

Chihuahua City, 31200, Mexico

Location

Investigational Site Number : 48405

Delegacion Benito Juarez, 03720, Mexico

Location

Investigational Site Number : 48404

Durango, 34000, Mexico

Location

Investigational Site Number : 48406

Mexico City, 06760, Mexico

Location

Investigational Site Number : 48403

Zapopan, 45030, Mexico

Location

Erasmus MC_Investigational Site Number 52801

Rotterdam, 3015 GD, Netherlands

Location

Investigational Site Number : 57802

Bergen, 5021, Norway

Location

Investigational Site Number : 57801

Grålum, 1714, Norway

Location

Investigational Site Number : 61617

Piła, Greater Poland Voivodeship, 64-920, Poland

Location

Investigational Site Number : 61609

Słupsk, Pomeranian Voivodeship, 76-200, Poland

Location

Investigational Site Number : 61615

Gdynia, 81-519, Poland

Location

Investigational Site Number : 61614

Poznan, 61 696, Poland

Location

Investigational Site Number : 61612

Warsaw, 02-776, Poland

Location

Investigational Site Number : 61613

Wroclaw, 50-556, Poland

Location

Investigational Site Number : 64307

Moscow, 119049, Russia

Location

Investigational Site Number : 64305

Moscow, 125167, Russia

Location

Investigational Site Number : 64304

Novosibirsk, 630090, Russia

Location

Investigational Site Number : 64301

Pyatigorsk, 357502, Russia

Location

Investigational Site Number : 64302

Saint Petersburg, 191024, Russia

Location

Investigational Site Number : 64306

Samara, 443099, Russia

Location

Investigational Site Number : 64303

Tula, 300053, Russia

Location

Investigational Site Number : 70201

Singapore, 119228, Singapore

Location

Investigational Site Number : 70202

Singapore, 169608, Singapore

Location

Investigational Site Number : 70203

Singapore, 308433, Singapore

Location

Investigational Site Number : 41004

Busan, Busan, 49241, South Korea

Location

Investigational Site Number : 41001

Suwon, Gyeonggi-do, 16499, South Korea

Location

Investigational Site Number : 41003

Seoul, Seoul-teukbyeolsi, 02841, South Korea

Location

Investigational Site Number : 41005

Seoul, Seoul-teukbyeolsi, 03080, South Korea

Location

Investigational Site Number : 41006

Seoul, Seoul-teukbyeolsi, 03722, South Korea

Location

Investigational Site Number : 72408

Málaga, Málaga, 29010, Spain

Location

Investigational Site Number : 72407

Valencia, Valenciana, Comunidad, 46010, Spain

Location

Investigational Site Number : 72412

Barcelona, 08035, Spain

Location

Investigational Site Number : 72414

Barcelona, 08035, Spain

Location

Investigational Site Number : 72409

Barcelona, 08041, Spain

Location

Investigational Site Number : 72416

Burgos, Spain

Location

Investigational Site Number : 72410

Madrid, 28007, Spain

Location

Investigational Site Number : 72411

Murcia, 30008, Spain

Location

Investigational Site Number : 72413

Seville, 41013, Spain

Location

Investigational Site Number : 76405

Bangkok, 10330, Thailand

Location

Investigational Site Number : 76404

Bangkok, 10400, Thailand

Location

Investigational Site Number : 76402

Chiang Mai, 50200, Thailand

Location

Investigational Site Number : 76401

Khon Kaen, 40002, Thailand

Location

Investigational Site Number : 76403

Songkhla, 90110, Thailand

Location

Investigational Site Number 79208

Ankara, 06620, Turkey (Türkiye)

Location

Investigational Site Number 79210

Istanbul, 34093, Turkey (Türkiye)

Location

Investigational Site Number 79206

Izmir, 35100, Turkey (Türkiye)

Location

Investigational Site Number 79209

Kayseri, 38039, Turkey (Türkiye)

Location

Investigational Site Number : 80408

Dnipropetrovsk, 49102, Ukraine

Location

Investigational Site Number : 80409

Kryvyi Rih, 50025, Ukraine

Location

Investigational Site Number : 80410

Kyiv, 03143, Ukraine

Location

Investigational Site Number : 82607

London, London, City of, W12 0HS, United Kingdom

Location

Investigational Site Number : 82604

Harrow, HA1 3UJ, United Kingdom

Location

Investigational Site Number : 82606

London, SE5 9PJ, United Kingdom

Location

Investigational Site Number : 82609

London, W2 1NY, United Kingdom

Location

Investigational Site Number : 82603

Manchester, M13 9WL, United Kingdom

Location

Investigational Site Number : 82605

Norfolk, NR31 6LA, United Kingdom

Location

Investigational Site Number : 82608

Southampton, SO16 6YD, United Kingdom

Location

Related Publications (3)

• 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.

  PMID: 40090011 DERIVED

• 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.

  PMID: 39899371 DERIVED

• 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.

  PMID: 37869360 DERIVED

Related Links

• EFC17093 Plain Language Results Summary

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

800-633-1610 ext 6#

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-Blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2020
• First Posted: September 24, 2020
• Study Start: December 14, 2020
• Primary Completion: January 15, 2025
• Study Completion (Estimated): August 26, 2026
• Last Updated: February 23, 2026
• Results First Posted: February 23, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

BR (7); FR (7); DE (4); AU (5); IL (5); JP (12); ME (6); SG (3); CA (3); CN (12); US (17); PL (6); RU (7); IT (6); KR (5); ES (9); TH (5); AR (5); HU (5); NL (1); TU (4); UA (3); GB (7); NO (2); CL (3)