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NCT04562766

PHASE3

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

Sponsor: Principia Biopharma, a Sanofi Company

View on ClinicalTrials.gov

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.

Official 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)

Key Details

Gender

All

Age Range

10 Years - Any

Study Type

INTERVENTIONAL

Enrollment

232

Start Date

2020-12-14

Completion Date

2026-09-02

Last Updated

2026-10-07

Healthy Volunteers

Conditions

Immune Thrombocytopenia

Interventions

DRUG

Rilzabrutinib

400mg Caplet

DRUG

Placebo

400mg Caplet

Inclusion Criteria: 1. Male and female with primary ITP with duration of \>6 months in pediatric participants aged 12 to \<18 years (pediatric participants aged 10 to \<12 years will be enrolled in the EU \[EEA countries\] only) and duration of \>3 months in ages 18 years and above 2. Patients who had a response (achievement of platelet count ≥50,000/µL) to IVIg/anti-D or CSs that was not sustained and who have documented intolerance, insufficient response or any contra-indication to any appropriate courses of standard of care ITP therapy 3. An average of 2 platelet counts at least 5 days apart of \<30,000/µL during the Screening period and no single platelet count \>35,000/µL, within 14 days prior to the first dose of study drug. \- Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator. 4. Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X 10\^9/L, AST/ALT ≤1.5 x upper limit of normal \[ULN\], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN \[unless the patient has documented Gilbert syndrome\], glomerular filtration rate \>50 \[Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants\]) 5. Hemoglobin \>9 g/dL within 1 week prior to Study Day 1 6. All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies 7. Patients must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the patient's guardian and agree to the schedule of assessments Exclusion Criteria: 1. Patients with secondary ITP 2. Pregnant or lactating women 3. History (within 5 years of Study Day 1) or current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non melanoma skin cancer 4. Transfusion with blood, blood products, plasmapheresis, or use of any other rescue medications with intent to increase platelet count within 14 days before Study Day 1 5. Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses) 6. Immunosuppressant drugs other than CSs within 5 times the elimination half-life of the drug or 14 days of Study Day 1, whichever is longer 7. Treatment with rituximab or splenectomy within the 3 months prior to Study Day 1 \- Patients treated with rituximab will have normal B-cell counts prior to enrollment 8. Had received any investigational drug within the 30 days before receiving the first dose of study medication, or at least 5 times elimination half-life of the drug (whichever is longer); patient should not be using an investigational device at the time of dosing * Patients who previously received treatment with Bruton's Tyrosine Kinase (BTK) inhibitors (except rilzabrutinib) within 30 days before the first dose of study drug are not eligible * Patients who previously received rilzabrutinib at any time are not eligible 9. History of solid organ transplant 10. Myelodysplastic syndrome 11. Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study 12. Planned surgery in the time frame of the dosing period

Locations (155)

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, Valparaiso, 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

And 55 more locations on ClinicalTrials.gov