Warm Autoimmune Hemolytic Anemia (wAIHA)Phase 2LUMINA 2NCT05002777
Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)
A Phase 2 study testing rilzabrutinib in people with Warm Autoimmune Hemolytic Anemia (wAIHA), run by Sanofi at 27 sites in 9 countries.
Trial summary
All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Warm Autoimmune Hemolytic Anemia (wAIHA)
- What is being tested?
- rilzabrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 27 sitesin 9 countries
- Study dates
- 7 Dec 2021to 31 Dec 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
- Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
- Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
- Up-to-date vaccination status as per local guidelines.
- Body mass index (BMI) >17.5 and <40 kg/m2
- All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
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- Core Part B
- Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A.
- Completion of Part A treatment period (24 weeks). Extended Part B
- Completion of Core Part B period.
Who cannot join
- 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.
- Secondary wAIHA from any cause including drugs, 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.
- Myelodysplastic syndrome.
- Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA.
- HIV infection.
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- Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start.
- Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent.
- Part B only
- Participants who receive any therapy during Part A known to be active in wAIHA.
- Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
27 sites in 9 countries.
27 sites in 9 countries
United States11 sites
Massachusetts General Hospital_Investigational Site Number: 8400001
Boston, Massachusetts, 02114
The Oncology Institute of Hope & Innovation Site Number : 8400006
Cerritos, California, 90703
TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006
Cerritos, California, 90703
TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006
Glendale, California, 91204
TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006
Long Beach, California, 90805
University of Southern California_Investigational Site Number: 8400009
Los Angeles, California, 90033
TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006
Santa Ana, California, 92705
Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002
Tamarac, Florida, 33321
The Lundquist Institute_Investigational Site Number: 8400005
Torrance, California, 90502
Georgetown University Hospital_Investigational Site Number: 8400003
Washington D.C., District of Columbia, 20007
TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006
Whittier, California, 90602
Spain4 sites
Hospital Universitario de Cruces_Investigational Site Number: 7240004
Barakaldo, 48093
Hospital Clinic de Barcelona_Investigational Site Number: 7240001
Barcelona, 08036
Hospital Universitario La Paz_Investigational Site Number: 7240003
Madrid, 28046
Hospital Universitario Virgen del Rocío_Investigational Site Number: 7240002
Seville, 41013
Italy3 sites
Ospedale Giuseppe Moscati_Investigational Site Number: 3800002
Avellino, 83100
Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori"_Investigational Site Number: 3800003
Meldola, 47014
Ospedale Maggiore Policlinico_Investigational Site Number: 3800001
Milan, 20149
United Kingdom3 sites
Leeds Teaching Hospitals NHS Trust_Investigational Site Number: 8260001
Leeds, LS9 7TF
Barts Health NHS Trust_Investigational Site Number: 8260005
London, E1 2ES
Imperial College Healthcare NHS Trust_Investigational Site Number: 8260002
London, W2 1NY
China2 sites
Peking Union Medical College Hospital_Investigational Site Number: 1560002
Beijing, 100005
Institute of hematology&blood diseases hospital_Investigational Site Number: 1560003
Tianjin, 300020
Austria1 site
Hanush-Krankenhaus_Investigational Site Number: 0400001
Vienna, 1140
Denmark1 site
Odense Universitetshospital Hæmatologisk Forskningsenhed_Investigational Site Number: 2080001
Odense, 5000
Germany1 site
Klinik für Hämatologie und Stammzellentransplantation_Investigational Site Number: 2760001
Essen, 45147
Hungary1 site
Fejer Varmegyei Szent Gyorgy Egyetemi Oktato Korhaz_Investigational Site Number: 3480001
Székesfehérvár, 8000
About this study
Official title: A Multicenter, Open-label, Phase IIb Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (LUMINA 2)
All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks.
There will be a 7-day safety follow-up period after receiving the last dose of study medication either in Part A (for those not eligible for Part B or early terminated) or Part B. In addition, each participant will be asked to attend an EOT-Core Part B visit when the last participant completes 52 weeks in Core Part B. The Extended Part B period will last for up to 253 weeks.
The study is currently in the Part B Extension segment.
- Conditions
- Warm Autoimmune Hemolytic Anemia (wAIHA)
- Being tested
- rilzabrutinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rilzabrutinibDrug
Also called PRN1008/SAR444671
Pharmaceutical form: Tablet Route of administration: Oral
| Study group | Role | What they receive |
|---|---|---|
| Rilzabrutinib | Experimental | Oral rilzabrutinib 400 mg BID Receives: Drug: rilzabrutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Proportion of participants with overall hemoglobin response
Response is defined as an increase in hemoglobin (Hb) by ≥2 g/dL from baseline and an absence of transfusion in the last 7 days, without biochemical resolution of hemolysis at the time response is achieved and an absence of rescue medications during the last 4 weeks.
Complete Response is defined as hemoglobin ≥11 g/dL (women) or ≥12 g/dL (men), no evidence of hemolysis (normal bilirubin, lactate dehydrogenase (LDH) , haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks.
Measured: By Week 24 in Part A
Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response
Durable response (Part B) is defined as Hb level ≥10 g/dL with an increase from baseline (Part A) of ≥2 g/dL on three consecutive scheduled visits during Week 24 to Week 50; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
Measured: By Week 50 in Part B
Secondary outcomes (5)
Proportion of participants with durable hemoglobin response
Durable response (Part A) is defined as Hb level ≥10 g/dL with an increase from baseline of ≥2 g/dL on three consecutive evaluable visits during the 24-week treatment period; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
Measured: By Week 24 in Part A
Median time from baseline to first hemoglobin response
Measured: From Day 1 to Week 24 in Part A
Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received
Measured: After Week 1 of treatment to Week 24 in Part A and Week 305 in Part B
Change from baseline in FACIT-Fatigue scale score
The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale is a 13-item questionnaire used to assesses self-reported fatigue and its impact on daily activities and function.
Measured: Until Week 24 in Part A and Week 305 in Part B
Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs)
Safety assessment including clinical laboratory evaluations, vital sign measurements and ECG
Measured: Until Week 24 in Part A and Week 305 in Part B
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2021
- Study start7 Dec 2021
- Primary completion23 May 2024
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Warm Autoimmune Hemolytic Anemia (wAIHA)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05002777
- Sponsor's protocol ID
- ACT17209
- Other IDs
- U1111-1262-2929, 2023-509441-13, 2021-001671-16, 2023
- Acronym
- LUMINA 2
- Status last verified
- Jun 2026
- First submitted
- 5 Aug 2021
- Last updated
- 5 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- 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
Publications
Cooper N, Kuter DJ, Frederiksen H, Han B, Chen F, Barcellini W, Fattizzo B, Cid J, Broome CM, Pampliega MV, Mazumder A, Lucchesi A, Zhao X, Risitano A, Sawhney S, Hill QA, Jayawardene D, Krenz H, Wang L, Zhao J, Daak A. Rilzabrutinib, an oral Bruton tyrosine kinase inhibitor, in patients with warm autoimmune haemolytic anaemia: a prospective, single-arm, multicentre, phase 2b safety and clinical activity study (LUMINA 2). Lancet Haematol. 2026 Oct;13(10):e698-e709. doi: 10.1016/S2352-3026(26)00218-8.
PubMed 42822477
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Phase IIb Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (LUMINA 2)
- ClinicalTrials.gov ID
- NCT05002777
- Acronym / sponsor ID
- LUMINA 2 / ACT17209
- Conditions
- Warm Autoimmune Hemolytic Anemia (wAIHA)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 22 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 7 Dec 2021 (actual)
- Primary completion
- 23 May 2024 (actual)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 12 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks.
There will be a 7-day safety follow-up period after receiving the last dose of study medication either in Part A (for those not eligible for Part B or early terminated) or Part B. In addition, each participant will be asked to attend an EOT-Core Part B visit when the last participant completes 52 weeks in Core Part B. The Extended Part B period will last for up to 253 weeks.
The study is currently in the Part B Extension segment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rilzabrutinib | Experimental | Drug: rilzabrutinibOral rilzabrutinib 400 mg BID |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
- Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
- Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
- Up-to-date vaccination status as per local guidelines.
- Body mass index (BMI) >17.5 and <40 kg/m2
Key exclusion criteria (12 listed)
- 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.
- Secondary wAIHA from any cause including drugs, 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.
- Myelodysplastic syndrome.
- Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rilzabrutinibAlso: PRN1008/SAR444671Pharmaceutical form: Tablet Route of administration: Oral | Drug | Rilzabrutinib |
Primary outcome
Part A: Proportion of participants with overall hemoglobin response
Time frame: By Week 24 in Part A
Response is defined as an increase in hemoglobin (Hb) by ≥2 g/dL from baseline and an absence of transfusion in the last 7 days, without biochemical resolution of hemolysis at the time response is achieved and an absence of rescue medications during the last 4 weeks. Complete Response is defined as hemoglobin ≥11 g/dL (women) or ≥12 g/dL (men), no evidence of hemolysis (normal bilirubin, lactate dehydrogenase (LDH) , haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks.
Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response
Time frame: By Week 50 in Part B
Durable response (Part B) is defined as Hb level ≥10 g/dL with an increase from baseline (Part A) of ≥2 g/dL on three consecutive scheduled visits during Week 24 to Week 50; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
5 secondary outcomes
Proportion of participants with durable hemoglobin response
Time frame: By Week 24 in Part A
Median time from baseline to first hemoglobin response
Time frame: From Day 1 to Week 24 in Part A
Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received
Time frame: After Week 1 of treatment to Week 24 in Part A and Week 305 in Part B
Change from baseline in FACIT-Fatigue scale score
Time frame: Until Week 24 in Part A and Week 305 in Part B
Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs)
Time frame: Until Week 24 in Part A and Week 305 in Part B
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2026.
- Derived from this study: PubMed 42822477Cooper N, Kuter DJ, Frederiksen H, Han B, Chen F, Barcellini W, Fattizzo B, Cid J, Broome CM, Pampliega MV, Mazumder A, Lucchesi A, Zhao X, Risitano A, Sawhney S, Hill QA, Jayawardene D, Krenz H, Wang L, Zhao J, Daak A. Rilzabrutinib, an oral Bruton tyrosine kinase inhibitor, in patients with warm autoimmune haemolytic anaemia: a prospective, single-arm, multicentre, phase 2b safety and clinical activity study (LUMINA 2). Lancet Haematol. 2026 Oct;13(10):e698-e709. doi: 10.1016/S2352-3026(26)00218-8.
- ACT17209 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
27 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rilzabrutinib in people with Warm Autoimmune Hemolytic Anemia (wAIHA), run by Sanofi at 27 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, Spain, Italy, United Kingdom, China and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Proportion of participants with overall hemoglobin response (time frame: By Week 24 in Part A). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05002777, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2026.