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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
Led by Sanofi · Updated on 2026-05-01
90
Participants Needed
92
Research Sites
227 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female participants with a confirmed diagnosis of primary warm autoimmune hemolytic anemia for at least 6 months.
- Participants who have failed to maintain response after corticosteroid treatment, are corticosteroid-dependent, intolerant, or ineligible.
- Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower.
- Use of contraception consistent with local regulations for clinical study participants.
You will not qualify if you...
- Clinically significant medical history or ongoing chronic illness that may jeopardize participant safety or study data quality.
- History of lymphoma, leukemia, or malignancy within the past 5 years, except certain resected skin cancers without metastasis for 3 years.
- Symptomatic herpes zoster within 3 months prior to screening.
- Mixed or secondary warm autoimmune hemolytic anemia from any cause, including drugs, Evans Syndrome, lymphoproliferative disorders (except low count monoclonal B-cell lymphocytosis), infectious or autoimmune diseases, or active hematologic malignancies.
- History of myelodysplastic syndrome.
- Uncontrolled or active HBV or active HCV infection.
- HIV infection.
- History of solid organ transplant.
- History of active or latent tuberculosis.
- Splenectomy within 12 weeks before screening or planned surgery during the primary analysis period.
- Use of other experimental drugs within 30 days or 5 half-lives prior to treatment.
- Previous treatment with BTK inhibitors for warm autoimmune hemolytic anemia before randomization.
- Sensitivity or allergy to study drugs or their components as judged by the investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 92 locations
1
Mayo Clinic in Arizona - Phoenix- Site Number : 8400032
Phoenix, Arizona, United States, 85054
Actively Recruiting
2
Noble Clinical Research- Site Number : 8400003
Tucson, Arizona, United States, 85704
Actively Recruiting
3
City of Hope National Medical Center- Site Number : 8400023
Duarte, California, United States, 91010
Actively Recruiting
4
USC Norris Comprehensive Cancer Center- Site Number : 8400007
Los Angeles, California, United States, 90033
Actively Recruiting
5
Stanford University Medical Center- Site Number : 8400026
Stanford, California, United States, 94305
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6
Lundquist Institute for Biomedical Innovation at Harbor - UCLA Medical Center- Site Number : 8400006
Torrance, California, United States, 90502
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7
Yale University School of Medicine- Site Number : 8400027
New Haven, Connecticut, United States, 06510
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8
Hialeah Hospital- Site Number : 8400009
Hialeah, Florida, United States, 33013
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9
Sylvester Comprehensive Cancer Center- Site Number : 8400031
Miami, Florida, United States, 33136
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10
University Hospital and Medical Center- Site Number : 8400005
Tamarac, Florida, United States, 33321
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11
University of Michigan Health System - Ann Arbor- Site Number : 8400014
Ann Arbor, Michigan, United States, 48109
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12
Henry Ford Hospital- Site Number : 8400012
Detroit, Michigan, United States, 48202
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13
Mayo Clinic in Rochester - Minnesota- Site Number : 8400008
Rochester, Minnesota, United States, 55905
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14
Montefiore Medical Center - Einstein Campus- Site Number : 8400011
The Bronx, New York, United States, 10461
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15
Ohio State University Hospital East- Site Number : 8400020
Columbus, Ohio, United States, 43203
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16
Fox Chase Cancer Center - Philadelphia- Site Number : 8400019
Philadelphia, Pennsylvania, United States, 19111
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17
Brown University Health - Rhode Island Hospital- Site Number : 8400028
Providence, Rhode Island, United States, 02903
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18
Baptist Memorial Hospital- Site Number : 8400018
Memphis, Tennessee, United States, 38120
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19
UT Health - San Antonio- Site Number : 8400015
San Antonio, Texas, United States, 78229
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20
Fred Hutchinson Cancer Center- Site Number : 8400016
Seattle, Washington, United States, 98109
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21
Gundersen Health System - La Crosse Medical Center- Site Number : 8400033
La Crosse, Wisconsin, United States, 54601
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22
Investigational Site Number : 0320001
Rosario, Santa Fe Province, Argentina, 2000
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23
Investigational Site Number : 0320003
Buenos Aires, Argentina, 1181
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24
Investigational Site Number : 0320002
Buenos Aires, Argentina, 1431
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25
Investigational Site Number : 0400001
Vienna, Austria, 1090
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26
Investigational Site Number : 0400002
Vienna, Austria, 1140
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27
Hemoes - Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos- Site Number : 0760002
Vitória, Espírito Santo, Brazil, 29047-105
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28
Universidade Federal de Goias- Site Number : 0760001
Goiânia, Goiás, Brazil, 74605-020
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29
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760003
São Paulo, Brazil, 05403-000
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30
Investigational Site Number : 1560001
Beijing, China, 100730
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31
Investigational Site Number : 1560007
Chengde, China, 067020
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32
Investigational Site Number : 1560011
Guangzhou, China, 510000
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33
Investigational Site Number : 1560005
Guangzhou, China, 510180
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34
Investigational Site Number : 1560009
Hohhot, China, 010050
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35
Investigational Site Number : 1560003
Nanchang, China, 330006
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36
Investigational Site Number : 1560012
Nanchang, China, 330006
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37
Investigational Site Number : 1560004
Suzhou, China, 215006
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38
Investigational Site Number : 1560002
Tianjin, China, 300020
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39
Investigational Site Number : 1560010
Wuhan, China, 430022
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40
Investigational Site Number : 1560006
Xi'an, China, 710054
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41
Investigational Site Number : 1560008
Zhengzhou, China, 450008
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42
Investigational Site Number : 2030002
Prague, Cardiff [Caerdydd Gb-crd], Czechia, 128 20
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43
Investigational Site Number : 2030001
Brno, Czechia, 625 00
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44
Investigational Site Number : 2080003
Aarhus, Denmark, 8200
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45
Investigational Site Number : 2080002
Copenhagen, Denmark, 2100
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46
Investigational Site Number : 2080001
Odense, Denmark, 5000
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47
Investigational Site Number : 2760003
Berlin, Germany, 10707
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48
Investigational Site Number : 2760001
Essen, Germany, 45147
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49
Investigational Site Number : 3000002
Athens, Greece, 115 27
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50
Investigational Site Number : 3000005
Larissa, Greece, 411 10
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51
Investigational Site Number : 3000004
Pátrai, Greece, 254 43
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52
Investigational Site Number : 3000003
Pátrai, Greece, 265 04
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53
Investigational Site Number : 3000001
Thessaloniki, Greece, 570 10
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54
Investigational Site Number : 3480002
Debrecen, Hungary, 4032
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55
Investigational Site Number : 3760001
Afula, Israel, 1834111
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56
Investigational Site Number : 3760007
Haifa, Israel, 3104802
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57
Investigational Site Number : 3760005
Kefar Sava, Israel, 4428164
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58
Investigational Site Number : 3760002
Tel Aviv, Israel, 6423906
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59
Investigational Site Number : 3800004
Florence, Firenze, Italy, 50134
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60
Investigational Site Number : 3800010
Genoa, Genova, Italy, 16132
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61
Investigational Site Number : 3800001
Milan, Milano, Italy, 20122
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62
Investigational Site Number : 3800005
Naples, Napoli, Italy, 80131
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63
Investigational Site Number : 3800002
Meldola, Reggio Emilia, Italy, 47014
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64
Investigational Site Number : 3800009
Novara, Italy, 28100
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65
Investigational Site Number : 3800006
Palermo, Italy, 90146
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66
Investigational Site Number : 3800007
Trieste, Italy, 34125
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67
Investigational Site Number : 3800008
Varese, Italy, 21100
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68
Investigational Site Number : 3800003
Vicenza, Italy, 36100
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69
Investigational Site Number : 3920007
Nagoya, Aichi-ken, Japan, 453-8511
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70
Investigational Site Number : 3920013
Tsuchiura, Ibaraki, Japan, 300-0028
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71
Investigational Site Number : 3920006
Kanazawa, Ishikawa-ken, Japan, 920-8201
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72
Investigational Site Number : 3920012
Fujisawa, Kanagawa, Japan, 251-0052
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73
Investigational Site Number : 3920003
Suita, Osaka, Japan, 565-0871
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74
Investigational Site Number : 3920001
Iruma, Saitama, Japan, 350-0495
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75
Investigational Site Number : 3920010
Kofu, Yamanashi, Japan, 400-0027
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76
Investigational Site Number : 3920015
Fukuoka, Japan, 815-8555
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77
Investigational Site Number : 3920002
Fukushima, Japan, 960-1295
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78
Investigational Site Number : 3920014
Kyoto, Japan, 605-0981
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79
Investigational Site Number : 3920008
Okayama, Japan, 700-8557
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80
Investigational Site Number : 3920004
Tokushima, Japan, 770-8503
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81
Investigational Site Number : 3920005
Yamagata, Japan, 990-9585
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82
Investigational Site Number : 5280002
Amsterdam, Netherlands, 1081 HV
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83
Investigational Site Number : 5280003
Leiden, Netherlands, 2333 ZA
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84
Investigational Site Number : 6160005
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-090
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85
Investigational Site Number : 6160006
Lodz, Lódzkie, Poland, 90-302
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86
Investigational Site Number : 7240005
Barakaldo, Basque Country, Spain, 48903
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87
Investigational Site Number : 7240001
Barcelona, Catalunya [Cataluña], Spain, 08036
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88
Investigational Site Number : 7240007
Majadahonda, Madrid, Spain, 28222
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89
Investigational Site Number : 7240003
Seville, Sevilla, Spain, 41013
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90
Investigational Site Number : 7240004
Madrid, Spain, 28007
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91
Investigational Site Number : 7520001
Huddinge, Sweden, 141 57
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92
Investigational Site Number : 7520003
Malmö, Sweden, 205 02
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Research Team
T
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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