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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.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

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.
Show all 12Show fewer
  • 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

Other trials in United States

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

Other trials in Spain

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

Other trials in Italy

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

Other trials in United Kingdom

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

Other trials in China

Austria1 site

  • Hanush-Krankenhaus_Investigational Site Number: 0400001

    Vienna, 1140

Other trials in Austria

Denmark1 site

  • Odense Universitetshospital Hæmatologisk Forskningsenhed_Investigational Site Number: 2080001

    Odense, 5000

Other trials in Denmark

Germany1 site

  • Klinik für Hämatologie und Stammzellentransplantation_Investigational Site Number: 2760001

    Essen, 45147

Other trials in Germany

Hungary1 site

  • Fejer Varmegyei Szent Gyorgy Egyetemi Oktato Korhaz_Investigational Site Number: 3480001

    Székesfehérvár, 8000

Other trials in Hungary

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.

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 groups in NCT05002777
Study groupRoleWhat they receive
RilzabrutinibExperimentalOral rilzabrutinib 400 mg BID
Receives: Drug: rilzabrutinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

  2. 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)

  1. 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

  2. Median time from baseline to first hemoglobin response

    Measured: From Day 1 to Week 24 in Part A

  3. 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

  4. 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

  5. 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.

  1. First posted12 Aug 2021
  2. Study start7 Dec 2021
  3. Primary completion23 May 2024
  4. Study completion31 Dec 2029 (estimated)

Resources

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)
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

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.

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