A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia
Start Date
8/18/2025
Completion Date
12/26/2029
Summary
This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb response (DHR) compared with placebo in approximately 90 male and female participants ≥ 18 years of age with a confirmed diagnosis of primary wAIHA. Following a 4-week screening period, eligible participants will be randomized in a 2:1 ratio to receive rilzabrutinib or placebo in primary analysis period (PAP) for a duration of up to 24 weeks. All participants who completed PAP will then continue in open-label period (OLP) to receive rilzabrutinib for a duration of 28 weeks. Upon the completion of OLP, only participants who demonstrate Hb increase during the last 8 weeks of OLP per specified criteria in the protocol will be eligible to continue in long-term extension (LTE) of the study. The duration of the LTE period will be from the first-participant-in (FPI)-LTE until the last participant completes 52 weeks in LTE. The safety follow-up period of this study following treatment completion or discontinuation will be 2 weeks.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
rilzabrutinib
placebo
Conditions
Locations
Mayo Clinic in Arizona - Phoenix- Site Number : 8400032
Phoenix, Arizona 85054
United States
Noble Clinical Research- Site Number : 8400003
Tucson, Arizona 85704
United States
City of Hope National Medical Center- Site Number : 8400023
Duarte, California 91010
United States
USC Norris Comprehensive Cancer Center- Site Number : 8400007
Los Angeles, California 90033
United States
Stanford University Medical Center- Site Number : 8400026
Stanford, California 94305
United States
Lundquist Institute for Biomedical Innovation at Harbor - UCLA Medical Center- Site Number : 8400006
Torrance, California 90502
United States
Yale University School of Medicine- Site Number : 8400027
New Haven, Connecticut 06510
United States
MedStar Georgetown University Hospital- Site Number : 8400004
Washington D.C., District of Columbia 20007
United States
Hialeah Hospital- Site Number : 8400009
Hialeah, Florida 33013
United States
Sylvester Comprehensive Cancer Center- Site Number : 8400031
Miami, Florida 33136
United States
University Hospital and Medical Center- Site Number : 8400005
Tamarac, Florida 33321
United States
Massachusetts General Hospital- Site Number : 8400001
Boston, Massachusetts 02114
United States
University of Michigan Health System - Ann Arbor- Site Number : 8400014
Ann Arbor, Michigan 48109
United States
Henry Ford Hospital- Site Number : 8400012
Detroit, Michigan 48202
United States
Mayo Clinic in Rochester - Minnesota- Site Number : 8400008
Rochester, Minnesota 55905
United States
Montefiore Medical Center - Einstein Campus- Site Number : 8400011
The Bronx, New York 10461
United States
Ohio State University Hospital East- Site Number : 8400020
Columbus, Ohio 43203
United States
Fox Chase Cancer Center - Philadelphia- Site Number : 8400019
Philadelphia, Pennsylvania 19111
United States
Brown University Health - Rhode Island Hospital- Site Number : 8400028
Providence, Rhode Island 02903
United States
Baptist Memorial Hospital- Site Number : 8400018
Memphis, Tennessee 38120
United States
UT Health - San Antonio- Site Number : 8400015
San Antonio, Texas 78229
United States
Fred Hutchinson Cancer Center- Site Number : 8400016
Seattle, Washington 98109
United States
Gundersen Health System - La Crosse Medical Center- Site Number : 8400033
La Crosse, Wisconsin 54601
United States
Investigational Site Number : 0320001
Rosario, Santa Fe Province 2000
Argentina
Investigational Site Number : 0320003
Buenos Aires, 1181
Argentina
Investigational Site Number : 0320002
Buenos Aires, 1431
Argentina
Investigational Site Number : 0400001
Vienna, 1090
Austria
Investigational Site Number : 0400002
Vienna, 1140
Austria
Hemoes - Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos- Site Number : 0760002
Vitória, Espírito Santo 29047-105
Brazil
Universidade Federal de Goias- Site Number : 0760001
Goiânia, Goiás 74605-020
Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760003
São Paulo, 05403-000
Brazil
Investigational Site Number : 1560001
Beijing, 100730
China
Investigational Site Number : 1560007
Chengde, 067020
China
Investigational Site Number : 1560011
Guangzhou, 510000
China
Investigational Site Number : 1560005
Guangzhou, 510180
China
Investigational Site Number : 1560009
Hohhot, 010050
China
Investigational Site Number : 1560003
Nanchang, 330006
China
Investigational Site Number : 1560012
Nanchang, 330006
China
Investigational Site Number : 1560004
Suzhou, 215006
China
Investigational Site Number : 1560002
Tianjin, 300020
China
Investigational Site Number : 1560010
Wuhan, 430022
China
Investigational Site Number : 1560006
Xi'an, 710054
China
Investigational Site Number : 1560008
Zhengzhou, 450008
China
Investigational Site Number : 2030002
Prague, Cardiff [Caerdydd Gb-crd] 128 20
Czechia
Investigational Site Number : 2030001
Brno, 625 00
Czechia
Investigational Site Number : 2080003
Aarhus, 8200
Denmark
Investigational Site Number : 2080002
Copenhagen, 2100
Denmark
Investigational Site Number : 2080001
Odense, 5000
Denmark
Investigational Site Number : 2760003
Berlin, 10707
Germany
Investigational Site Number : 2760001
Essen, 45147
Germany
Investigational Site Number : 3000002
Athens, 115 27
Greece
Investigational Site Number : 3000005
Larissa, 411 10
Greece
Investigational Site Number : 3000004
Pátrai, 254 43
Greece
Investigational Site Number : 3000003
Pátrai, 265 04
Greece
Investigational Site Number : 3000001
Thessaloniki, 570 10
Greece
Investigational Site Number : 3480004
Budapest, 1085
Hungary
Investigational Site Number : 3480002
Debrecen, 4032
Hungary
Investigational Site Number : 3760001
Afula, 1834111
Israel
Investigational Site Number : 3760007
Haifa, 3104802
Israel
Investigational Site Number : 3760005
Kefar Sava, 4428164
Israel
Investigational Site Number : 3760002
Tel Aviv, 6423906
Israel
Investigational Site Number : 3800004
Florence, Firenze 50134
Italy
Investigational Site Number : 3800010
Genoa, Genova 16132
Italy
Investigational Site Number : 3800001
Milan, Milano 20122
Italy
Investigational Site Number : 3800005
Naples, Napoli 80131
Italy
Investigational Site Number : 3800002
Meldola, Reggio Emilia 47014
Italy
Investigational Site Number : 3800009
Novara, 28100
Italy
Investigational Site Number : 3800006
Palermo, 90146
Italy
Investigational Site Number : 3800007
Trieste, 34125
Italy
Investigational Site Number : 3800008
Varese, 21100
Italy
Investigational Site Number : 3800003
Vicenza, 36100
Italy
Investigational Site Number : 3920007
Nagoya, Aichi-ken 453-8511
Japan
Investigational Site Number : 3920013
Tsuchiura, Ibaraki 300-0028
Japan
Investigational Site Number : 3920006
Kanazawa, Ishikawa-ken 920-8201
Japan
Investigational Site Number : 3920012
Fujisawa, Kanagawa 251-0052
Japan
Investigational Site Number : 3920003
Suita, Osaka 565-0871
Japan
Investigational Site Number : 3920001
Iruma, Saitama 350-0495
Japan
Investigational Site Number : 3920010
Kofu, Yamanashi 400-0027
Japan
Investigational Site Number : 3920015
Fukuoka, 815-8555
Japan
Investigational Site Number : 3920002
Fukushima, 960-1295
Japan
Investigational Site Number : 3920014
Kyoto, 605-0981
Japan
Investigational Site Number : 3920008
Okayama, 700-8557
Japan
Investigational Site Number : 3920004
Tokushima, 770-8503
Japan
Investigational Site Number : 3920005
Yamagata, 990-9585
Japan
Investigational Site Number : 5280002
Amsterdam, 1081 HV
Netherlands
Investigational Site Number : 5280003
Leiden, 2333 ZA
Netherlands
Investigational Site Number : 6160005
Bydgoszcz, Kuyavian-Pomeranian Voivodeship 85-090
Poland
Investigational Site Number : 6160006
Lodz, Lódzkie 90-302
Poland
Investigational Site Number : 7240005
Barakaldo, Basque Country 48903
Spain
Investigational Site Number : 7240001
Barcelona, Catalunya [Cataluña] 08036
Spain
Investigational Site Number : 7240007
Majadahonda, Madrid 28222
Spain
Investigational Site Number : 7240003
Seville, Sevilla 41013
Spain
Investigational Site Number : 7240004
Madrid, 28007
Spain
Investigational Site Number : 7520001
Huddinge, 141 57
Sweden
Investigational Site Number : 7520003
Malmö, 205 02
Sweden