Study of rilzabrutinib tablets compared to placebo in adults with warm autoimmune hemolytic anemia

3 1

What is this study about?

This clinical study focuses on warm autoimmune hemolytic anemia (wAIHA), a condition where the body’s immune system mistakenly destroys its own red blood cells. The study will test a medication called rilzabrutinib, given as film-coated tablets taken by mouth, compared to a placebo. This is a new treatment approach for people whose disease has not responded well to standard treatments or who cannot take conventional medications.

The research aims to determine if rilzabrutinib can help maintain stable blood levels in people with warm autoimmune hemolytic anemia. During the study, participants will receive either rilzabrutinib tablets or placebo tablets. The maximum daily dose of rilzabrutinib will be 800 mg, and the treatment period will last for 224 weeks.

The study will measure several aspects of the treatment’s effects, including changes in hemoglobin levels (a protein in red blood cells that carries oxygen throughout the body), changes in fatigue levels, and how quickly the treatment works. The study will also track how well participants feel, including their ability to breathe comfortably, and monitor any side effects that may occur during treatment.

1 Initial participation

After confirming your eligibility for the study, you will be diagnosed with warm autoimmune hemolytic anemia (wAIHA) for at least 3 months

Your physical condition will be evaluated using the Eastern Cooperative Oncology Group (ECOG) scale, which must be Grade 2 or lower

You must have previously tried corticosteroid treatment without maintaining a sustained response, or be unable to take corticosteroids

2 Treatment assignment

You will be randomly assigned to receive either rilzabrutinib tablets or placebo tablets

The tablets are to be taken by mouth

Neither you nor your doctor will know which treatment you are receiving during this phase

3 Primary assessment period

Your hemoglobin levels will be regularly monitored to track your response to treatment

Your fatigue levels will be measured using a special questionnaire called FACIT-fatigue

Your breathing difficulties will be assessed using FACIT-Dyspnea questionnaire

Blood tests will check your LDH (lactate dehydrogenase) levels

4 Ongoing monitoring

Regular physical examinations will be conducted

Your vital signs will be checked periodically

You will undergo heart monitoring through electrocardiograms (ECG)

Any side effects or health changes will be recorded and monitored

5 Study duration

The study is scheduled to run from June 2025 to October 2029

After the initial double-blind period, there will be an open-label period and long-term extension phase

Who Can Join the Study?

Must be diagnosed with primary warm autoimmune hemolytic anemia (wAIHA) for at least 3 months (a condition where the body’s immune system destroys red blood cells)
Must have experienced one of these situations with corticosteroid treatment (medications like prednisone that reduce inflammation):
– Failed to respond to treatment within 3 weeks while taking at least 1 mg/kg or 60 mg daily
– Need more than 10 mg daily to maintain improvement
– Cannot tolerate the medication due to side effects
– Have medical conditions that make corticosteroid use unsafe
Must have an ECOG performance status of 2 or lower (ability to carry out daily activities – Grade 2 means able to walk and handle self-care but unable to work)
Both men and women can participate
Must use appropriate contraception methods during the study according to local guidelines
Must be an adult (18 years or older)

Who Cannot Join the Study?

Current diagnosis of Evans syndrome (a condition where the body makes antibodies against both red blood cells and platelets)
History of blood clotting disorders or significant blood clots in the past 6 months
Recent major surgery within 4 weeks before starting the study
Active or chronic hepatitis B or C infection (liver infections)
Current HIV infection
Active tuberculosis infection
Presence of any active cancer or history of cancer within the past 3 years (except for successfully treated skin cancer)
Severe heart conditions or uncontrolled high blood pressure
Severe kidney disease requiring dialysis
Current pregnancy or breastfeeding
Use of certain medications that affect blood clotting within 7 days before starting the study
Participation in another clinical trial within 30 days before this study
Allergic reactions to similar medications in the past
Any condition that, in the opinion of the study doctor, would make participation unsafe

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Mazowiecki Szpital Specjalistyczny w Radomiu	Radom	Poland
Medical University Of Vienna	Vienna	Austria
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy

Other Sites

Site Name	City	Country
Azienda Sanitaria Universitaria Giuliano Isontina	Trieste	Italy
Institute Of Hematology And Blood Transfusion	Prague	Czechia
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Azienda Unita Locale Socio Sanitaria N 8 Berica	Vicenza	Italy
Geniko Nosokomeio Thessalonikis George Papanikolaou	Thessaloniki	Greece
Santa Sp. z o.o.	Lodz	Poland
Azienda Socio Sanitaria Territoriale Dei Sette Laghi	Varese	Italy
Region Skane Skanes Universitetssjukhus	Lund	Sweden
General University Hospital Of Larissa	Larissa	Greece
University Of Debrecen	Debrecen	Hungary
Virgen del Rocío University Hospital	Sevilla	Spain
Odense University Hospital	Odense	Denmark
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
Fakultni Nemocnice Brno	Brno	Czechia
Karolinska University Hospital	Solna	Sweden
Semmelweis University	Budapest	Hungary
Hospital Universitario De Cruces	Barakaldo	Spain
IRCCS Ospedale Policlinico San Martino	Genoa	Italy
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse	Vienna	Austria
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet	Budapest	Hungary
General University Hospital Of Patras	Patras	Greece
In Vivo Sp. z o.o.	Bydgoszcz	Poland
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Region Midtjylland	Aarhus	Denmark
Rigshospitalet	Copenhagen	Denmark
Olympion Therapeftirio General Clinic Of Patras S.A.	Patras	Greece
Onkologische Schwerpunktpraxis (OSP) Kurfürstendamm	Berlin	Germany
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Uoohqwaztuwoybzejtcjm Emghd Atd	Essen	Germany
Lomzx Ugjyiydkxtvy Mgvgrxo Cqizltw (ymtta	Leiden	The Netherlands
Lgzqs Gvmmnrq Hxsplrul Ov Afituu	Athens	Greece
Abwuunp Oppxasjghpq Ogureyfl Rvhsgpq Vbdmc Sileb Civqbkwh	Palermo	Italy
Ilidymzs Rpsylzadt Pqu Lh Smipjc Dty Ttsyxh Djwx Azvmzkl Ilka Suvhcf	Meldola	Italy
Aoobuigpn Ueg	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
Austria	Recruiting	15.06.2025
Czechia	Recruiting	15.06.2025
Denmark	Not yet recruiting	15.06.2025
Germany	Recruiting	15.06.2025
Greece	Recruiting	15.06.2025
Hungary	Recruiting	15.06.2025
Italy	Recruiting	15.06.2025
Poland	Recruiting	15.06.2025
Spain	Recruiting	15.06.2025
Sweden	Recruiting	15.06.2025
The Netherlands	Recruiting	15.06.2025

Trial locations

Investigated drugs:

Rilzabrutinib

Rilzabrutinib is a medication being studied for treating warm autoimmune hemolytic anemia (wAIHA), a condition where the body’s immune system destroys its own red blood cells. This medication works by targeting and inhibiting an enzyme called BTK (Bruton’s tyrosine kinase), which plays a role in the immune system response. The goal is to reduce the destruction of red blood cells and help maintain healthy hemoglobin levels in patients with this condition.

Autoimmune Hemolytic Anemia – A rare condition where the immune system mistakenly attacks and destroys red blood cells. The destruction of red blood cells occurs faster than the body can replace them, leading to anemia. The condition causes the body to produce antibodies that attach to red blood cells, marking them for destruction by the immune system. This process takes place primarily in the spleen and liver, where damaged red blood cells are broken down. The condition can develop gradually or suddenly, and can occur at any age.

Trial ID:

2024-517972-39-00

Protocol code:

EFC17360 – LUMINA 3

Trial Phase:

Therapeutic confirmatory (Phase III)

Other Trials to Consider