Autoimmune Haemolytic AnaemiaPhase 3LUMINA 3NCT07086976
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
A Phase 3 study testing rilzabrutinib in people with Autoimmune Haemolytic Anaemia, run by Sanofi at 105 sites in 18 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Haemolytic Anaemia
- What is being tested?
- rilzabrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 105 sitesin 18 countries
- Study dates
- 18 Aug 2025to 26 Dec 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female participants with a documented (confirmed) diagnosis of primary wAIHA for at least 6 months.
- Participants who have previously failed to maintain a sustained response after treatment with CS (CS-resistance [defined as failure to obtain hemoglobin response within 3 weeks on at least 1 mg/kg or 60 mg prednisone or equivalent per day], CS-dependent wAIHA [defined as need to continue on prednisone or equivalent at a dose of >10 mg/day to maintain a response]), or are intolerant or ineligible to CS (defined as with contraindications, pre-existing medical conditions or CS-related complications that may render CS intolerant or ineligible per the best clinical judgement of the investigators).
- Participants with Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower.
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Who cannot join
- Participants with clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
- Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
- Participants with symptomatic herpes zoster within 3 months prior to screening.
- Participants with mixed wAIHA, or secondary wAIHA from any cause including drugs, Evans Syndrome, lymphoproliferative disorders (low count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
- Participants with history of myelodysplastic syndrome.
- Participants with uncontrolled or active HBV infection or Active HCV infection.
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- HIV infection.
- Participants with history of solid organ transplant.
- Participants with a history of active or latent tuberculosis (TB).
- Splenectomy within 12 weeks before screening and planned surgery during the PAP.
- Concurrent treatment with other experimental/investigational drugs within 30 days or 5 half-lives, whichever is longer, prior to treatment start. Participants who previously received treatment with BTK inhibitors for wAIHA before Day 1 (randomization) are not eligible.
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
- The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial locations and contacts
105 sites in 18 countries. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free for US & Canada) contact
105 sites in 18 countries
United States27 sites, 27 recruiting
- Recruiting
University of Michigan Health System - Ann Arbor- Site Number : 8400014
Ann Arbor, Michigan, 48109
- Recruiting
Massachusetts General Hospital- Site Number : 8400001
Boston, Massachusetts, 02114
- Recruiting
Beth Israel Deaconess Medical Center - Boston- Site Number : 8400034
Boston, Massachusetts, 02215
- Recruiting
Cleveland Clinic - Cleveland- Site Number : 8400029
Cleveland, Ohio, 44195
- Recruiting
Ohio State University Hospital East- Site Number : 8400020
Columbus, Ohio, 43203
- Recruiting
Henry Ford Hospital- Site Number : 8400012
Detroit, Michigan, 48202
- Recruiting
City of Hope Comprehensive Cancer Center- Site Number : 8400023
Duarte, California, 91010
- Recruiting
Hialeah Hospital- Site Number : 8400009
Hialeah, Florida, 33013
- Recruiting
Gundersen Health System - La Crosse Medical Center- Site Number : 8400033
La Crosse, Wisconsin, 54601
- Recruiting
Cancer and Blood Specialty Clinic - Los Alamitos- Site Number : 8400038
Los Alamitos, California, 90720
- Recruiting
USC Norris Comprehensive Cancer Center- Site Number : 8400007
Los Angeles, California, 90033
- Recruiting
Baptist Memorial Hospital- Site Number : 8400018
Memphis, Tennessee, 38120
- Recruiting
Sylvester Comprehensive Cancer Center- Site Number : 8400031
Miami, Florida, 33136
- Recruiting
Yale University School of Medicine- Site Number : 8400027
New Haven, Connecticut, 06510
- Recruiting
Tulane University School of Medicine- Site Number : 8400037
New Orleans, Louisiana, 70112
- Recruiting
Fox Chase Cancer Center - Philadelphia- Site Number : 8400019
Philadelphia, Pennsylvania, 19111
- Recruiting
Mayo Clinic in Arizona - Phoenix- Site Number : 8400032
Phoenix, Arizona, 85054
- Recruiting
Brown University Health - Rhode Island Hospital- Site Number : 8400028
Providence, Rhode Island, 02903
- Recruiting
Mayo Clinic in Rochester - Minnesota- Site Number : 8400008
Rochester, Minnesota, 55905
- Recruiting
UT Health - San Antonio- Site Number : 8400015
San Antonio, Texas, 78229
- Recruiting
Fred Hutchinson Cancer Center- Site Number : 8400016
Seattle, Washington, 98109
- Recruiting
Stanford University Medical Center- Site Number : 8400026
Stanford, California, 94305
- Recruiting
University Hospital and Medical Center- Site Number : 8400005
Tamarac, Florida, 33321
- Recruiting
Montefiore Medical Center - Einstein Campus- Site Number : 8400011
The Bronx, New York, 10461
- Recruiting
Lundquist Institute for Biomedical Innovation at Harbor - UCLA Medical Center- Site Number : 8400006
Torrance, California, 90502
- Recruiting
Noble Clinical Research- Site Number : 8400003
Tucson, Arizona, 85704
- Recruiting
MedStar Georgetown University Hospital- Site Number : 8400004
Washington D.C., District of Columbia, 20007
Japan13 sites, 13 recruiting
- Recruiting
Investigational Site Number : 3920012
Fujisawa, Kanagawa, 251-0052
- Recruiting
Investigational Site Number : 3920015
Fukuoka, 815-8555
- Recruiting
Investigational Site Number : 3920002
Fukushima, 960-1295
- Recruiting
Investigational Site Number : 3920001
Iruma, Saitama, 350-0495
- Recruiting
Investigational Site Number : 3920006
Kanazawa, Ishikawa-ken, 920-8201
- Recruiting
Investigational Site Number : 3920010
Kofu, Yamanashi, 400-0027
- Recruiting
Investigational Site Number : 3920014
Kyoto, 605-0981
- Recruiting
Investigational Site Number : 3920007
Nagoya, Aichi-ken, 453-8511
- Recruiting
Investigational Site Number : 3920008
Okayama, 700-8557
- Recruiting
Investigational Site Number : 3920003
Suita, Osaka, 565-0871
- Recruiting
Investigational Site Number : 3920004
Tokushima, 770-8503
- Recruiting
Investigational Site Number : 3920013
Tsuchiura, Ibaraki, 300-0028
- Recruiting
Investigational Site Number : 3920005
Yamagata, 990-9585
China12 sites, 12 recruiting
- Recruiting
Investigational Site Number : 1560001
Beijing, 100730
- Recruiting
Investigational Site Number : 1560007
Chengde, 067020
- Recruiting
Investigational Site Number : 1560011
Guangzhou, 510000
- Recruiting
Investigational Site Number : 1560005
Guangzhou, 510180
- Recruiting
Investigational Site Number : 1560009
Hohhot, 010050
- Recruiting
Investigational Site Number : 1560003
Nanchang, 330006
- Recruiting
Investigational Site Number : 1560012
Nanchang, 330006
- Recruiting
Investigational Site Number : 1560004
Suzhou, 215006
- Recruiting
Investigational Site Number : 1560002
Tianjin, 300020
- Recruiting
Investigational Site Number : 1560010
Wuhan, 430022
- Recruiting
Investigational Site Number : 1560006
Xi'an, 710054
- Recruiting
Investigational Site Number : 1560008
Zhengzhou, 450008
Italy10 sites, 10 recruiting
- Recruiting
Investigational Site Number : 3800004
Florence, Firenze, 50134
- Recruiting
Investigational Site Number : 3800010
Genoa, Genova, 16132
- Recruiting
Investigational Site Number : 3800002
Meldola, Reggio Emilia, 47014
- Recruiting
Investigational Site Number : 3800001
Milan, Milano, 20122
- Recruiting
Investigational Site Number : 3800005
Naples, Napoli, 80131
- Recruiting
Investigational Site Number : 3800009
Novara, 28100
- Recruiting
Investigational Site Number : 3800006
Palermo, 90146
- Recruiting
Investigational Site Number : 3800007
Trieste, 34125
- Recruiting
Investigational Site Number : 3800008
Varese, 21100
- Recruiting
Investigational Site Number : 3800003
Vicenza, 36100
Greece5 sites, 5 recruiting
- Recruiting
Investigational Site Number : 3000002
Athens, 115 27
- Recruiting
Investigational Site Number : 3000001
Exochi, 570 10
- Recruiting
Investigational Site Number : 3000005
Larissa, 411 10
- Recruiting
Investigational Site Number : 3000004
Pátrai, 254 43
- Recruiting
Investigational Site Number : 3000003
Pátrai, 265 04
Israel5 sites, 5 recruiting
- Recruiting
Investigational Site Number : 3760001
Afula, 1834111
- Recruiting
Investigational Site Number : 3760007
Haifa, 3104802
- Recruiting
Investigational Site Number : 3760005
Kefar Sava, 4428164
- Recruiting
Investigational Site Number : 3760008
Safed, 1311001
- Recruiting
Investigational Site Number : 3760002
Tel Aviv, 6423906
Spain5 sites, 5 recruiting
- Recruiting
Investigational Site Number : 7240001
Barcelona, Catalunya [Cataluña], 08036
- Recruiting
Investigational Site Number : 7240005
Bizkaia, Basque Country, 48903
- Recruiting
Investigational Site Number : 7240004
Madrid, 28007
- Recruiting
Investigational Site Number : 7240007
Majadahonda, Madrid, 28222
- Recruiting
Investigational Site Number : 7240003
Seville, Andalusia, 41013
Germany4 sites, 4 recruiting
- Recruiting
Investigational Site Number : 2760003
Berlin, 10707
- Recruiting
Investigational Site Number : 2760001
Essen, 45147
- Recruiting
Investigational Site Number : 2760004
Frankfurt, 60389
- Recruiting
Investigational Site Number : 2760006
Leipzig, 04103
Argentina3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 0320003
Buenos Aires, 1181
- Recruiting
Investigational Site Number : 0320002
Buenos Aires, 1431
- Recruiting
Investigational Site Number : 0320001
Rosario, Santa Fe Province, 2000
Brazil3 sites, 3 recruiting
- Recruiting
Universidade Federal de Goias- Site Number : 0760001
Goiânia, Goiás, 74605-020
- Recruiting
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760003
São Paulo, 05403-000
- Recruiting
Hemoes - Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos- Site Number : 0760002
Vitória, Espírito Santo, 29047-105
Denmark3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 2080003
Aarhus, 8200
- Recruiting
Investigational Site Number : 2080002
Copenhagen, 2100
- Recruiting
Investigational Site Number : 2080001
Odense, 5000
Sweden3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 7520004
Gothenburg, 413 45
- Recruiting
Investigational Site Number : 7520001
Huddinge, 141 57
- Recruiting
Investigational Site Number : 7520003
Malmö, 205 02
Austria2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 0400001
Vienna, 1090
- Recruiting
Investigational Site Number : 0400002
Vienna, 1140
Czechia2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 2030001
Brno, 625 00
- Recruiting
Investigational Site Number : 2030002
Prague, 128 20
Hungary2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 3480004
Budapest, 1085
- Recruiting
Investigational Site Number : 3480002
Debrecen, 4032
Netherlands2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 5280002
Amsterdam, 1081 HV
- Recruiting
Investigational Site Number : 5280003
Leiden, 2333 ZA
Poland2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 6160005
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-090
- Recruiting
Investigational Site Number : 6160006
Lodz, Lódzkie, 90-302
Saudi Arabia2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 6820004
Jeddah, 23433
- Recruiting
Investigational Site Number : 6820003
Riyadh, 12372
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study With an Open-label Period and Long-term Extension to Assess the Efficacy and Safety of Rilzabrutinib in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)
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
- Autoimmune Haemolytic Anaemia
- Being tested
- rilzabrutinib, placebo
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rilzabrutinibDrug
Also called SAR444671
Pharmaceutical form:tablet-Route of administration:oral
placeboDrug
Pharmaceutical form:tablet-Route of administration:oral
| Study group | Role | What they receive |
|---|---|---|
| rilzabrutinib | Experimental | Oral rilzabrutinib BID Receives: Drug: rilzabrutinib |
| placebo | Placebo comparator | Oral placebo BID Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants achieving DHR. DHR is defined as an increase of Hb by ≥2 g/dL from baseline on at least two thirds of evaluable scheduled visits between Week 12 and Week 24 (inclusive) in the PAP
DHR is defined as an increase of Hb by ≥2 g/dL from baseline on at least two thirds of evaluable scheduled visits between Week 12 and Week 24 (inclusive) in the PAP in the absence of rescue medication and transfusion.
Measured: By Week 24
Secondary outcomes (7)
Proportion of participants achieving overall Hb response (response [R] or complete response [CR]) by Week 24 of treatment in the PAP
Response is defined as an increase in Hb by ≥2 g/dL from baseline in the absence of transfusion and rescue medication.
Complete Response is defined as Hb ≥12 g/dL (women) or ≥13 g/dL (men) without evidence of hemolysis (ie, normal indirect bilirubin, lactate dehydrogenase [LDH], haptoglobin, and reticulocytes), in the absence of transfusion and rescue medication.
Measured: By Week 24
Change from baseline in fatigue total score as measured by Functional Assessment of Chronic Illness Therapy (FACIT) - fatigue at Week 24
Measured: Baseline to Week 24
The time taken (days) to achieve the first Hb increase by ≥2 g/dL from baseline during the PAP in the previous 14 days and in the absence of rescue medication in the previous 6 weeks
Measured: Until Week 24
Change from baseline in levels of LDH at Week 24
Measured: Baseline to Week 24
Proportion of participants requiring use of rescue therapy after Week 4 of treatment during the PAP
Measured: Until Week 24
Change from baseline in dyspnea severity score as measured by FACIT-Dyspnea at Week 24
Measured: Baseline to Week 24
Incidence of treatment-emergent adverse events (TEAE), treatment-emergent serious adverse events (SAEs), treatment-emergent adverse events of special interest, as well as clinical laboratory evaluations, vital sign, physical exam, and electrocardiograms
Measured: Until Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2025
- Study start18 Aug 2025
- Primary completion31 May 2028 (estimated)
- Study completion26 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune Haemolytic AnaemiaStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07086976
- Sponsor's protocol ID
- EFC17360
- Other IDs
- U1111-1314-5112, 2024-517972-39
- Acronym
- LUMINA 3
- Status last verified
- Oct 2026
- First submitted
- 17 Jul 2025
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org