Study of oral rilzabrutinib for adults with immune thrombocytopenia who did not respond to first-line treatment

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What is this study about?

This study focuses on people with immune thrombocytopenia (ITP), a condition where the immune system mistakenly attacks and destroys blood platelets, leading to low platelet counts and increased risk of bleeding. The study will test a medication called rilzabrutinib, which is taken by mouth in tablet form, in adults whose ITP has not improved with initial treatments such as corticosteroids or immunoglobulins.

The purpose of this research is to determine if rilzabrutinib can help maintain higher platelet counts over an extended period in people with ITP. During the study, participants will take rilzabrutinib tablets daily for approximately 80 weeks. The maximum daily dose will be 800 mg.

Throughout the study, participants will have their platelet counts checked regularly to monitor how well the treatment is working. The study will also look at whether the treatment can reduce bleeding problems and allow participants to decrease or stop their corticosteroid medications. Participants’ blood will be tested to check their overall health, including liver and kidney function.

1 Initial visit and medication start

You will begin taking rilzabrutinib tablets orally. This medication is being tested for immune thrombocytopenia (ITP), a condition affecting blood platelet levels.

Your initial platelet count will be measured to establish a baseline.

2 Regular monitoring – First 4 weeks

Your platelet count will be checked regularly to monitor for response to treatment.

A positive response is indicated by achieving a platelet count of at least 50,000/μL, or between 30,000/μL and 50,000/μL with at least double your starting count.

These measurements must be recorded at least 5 days apart.

3 Ongoing monitoring – Through week 28

Platelet counts will continue to be checked every 2 weeks.

Your bleeding symptoms will be evaluated using a standardized scale.

If you are taking corticosteroids, your dose may be adjusted based on your response to treatment.

4 Final evaluation period – Last 12 weeks

During the final 12 weeks, you must maintain the target platelet levels for at least half of your scheduled check-ups.

A minimum of 4 valid visits showing maintained platelet counts is required.

Your response will be considered successful if you maintain these levels without requiring emergency treatment.

5 Study completion

The study will evaluate if the medication provided a lasting increase in your platelet count.

Final assessments will include blood tests and evaluation of any changes in bleeding symptoms.

Your corticosteroid use will be reviewed to determine if the dose was reduced by 50% or to less than 5 mg.

Who Can Join the Study?

  • Must be 18 years or older when signing the consent form
  • Must have confirmed diagnosis of primary immune thrombocytopenia (ITP) – a condition where the immune system attacks blood platelets
  • Must have an ECOG performance status of 2 or lower – meaning able to carry out most daily activities with minimal assistance
  • Must have received at least one first-line therapy such as corticosteroids, intravenous immunoglobulin, or anti-D treatment
  • Must have shown improvement in platelet count (above 30,000 per microliter) during previous treatment
  • Must have up-to-date vaccinations according to local guidelines
  • Must use appropriate contraception methods if sexually active
  • Must have adequate blood test results showing:
    • Hemoglobin above 9 g/dL
    • Adequate white blood cell count
    • Normal liver function tests
    • Normal kidney function
    • Normal blood clotting tests
  • Must be able to understand and sign the informed consent form

Who Cannot Join the Study?

  • Age below 18 years or above 65 years
  • Currently pregnant or breastfeeding women
  • History of blood clots or thrombotic events (formation of blood clots in blood vessels)
  • Active bleeding or high risk of bleeding
  • Recent major surgery within the last 30 days
  • Current participation in other clinical trials
  • Severe liver problems or abnormal liver function tests
  • Severe kidney problems or impaired kidney function
  • History of cancer in the past 5 years
  • Uncontrolled high blood pressure
  • Heart problems including heart failure (when heart cannot pump blood effectively)
  • Active infections including hepatitis B, hepatitis C, or HIV
  • History of autoimmune disorders (conditions where immune system attacks healthy cells) other than ITP
  • Regular use of blood thinners or anticoagulation medications
  • Alcohol or substance abuse within the past year

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Azienda Sanitaria Universitaria Giuliano Isontina Trieste Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
ASST Grande Ospedale Metropolitano Niguarda Milan Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse Vienna Austria
Justus-Liebig-Universitaet Giessen Giessen Germany
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital General Universitario Morales Meseguer Murcia Spain
Hospital Del Mar Barcelona Spain
Azienda Unita Locale Socio Sanitaria N 8 Berica Vicenza Italy
Copernicus Podmiot Leczniczy Sp. z o.o. Gdansk Poland
Wojewodzki Szpital Specjalistyczny Im. Janusza Korczaka W Slupsku Sp. z o.o. Slupsk Poland
Somogy Varmegyei Kaposi Mor Oktato Korhaz Kaposvar Hungary
Virgen del Rocío University Hospital Sevilla Spain
Hospital Universitario De Canarias La Laguna Spain
Semmelweis University Budapest Hungary
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Hopital Beaujon Clichy France
Area De Salud De Burgos Y Soria Burgos Spain
University Of Debrecen Debrecen Hungary
Hopital Prive D Antony Antony France
Servei De Salut De Les Illes Balears Palma Spain
University Hospital Ostrava Ostrava Czechia
Mpw Lywqmwa Mufqm Leipzig Germany
Ckvbnb Hxnzntrsqvc Uqfbrtywgejgo Ds Dxpuy Dijon France
Imecxcml Rimbcmhth Pwb Ld Sdskri Dub Tugsra Diku Asmzkzj Imbv Sbgbbc Meldola Italy
Ajzvndy Ozwsxgmvgmn Uikkzctpormmz Celufmcxonqy Dyrgp Sflxne E Dcgha Slymrpc Dq Tmjpvm Turin Italy
Gxmptc Ulsfsecyxd Fsqcvumud Frankfurt Germany
Uhqynlreagerlhndebxql Eikhy Akm Essen Germany
Wbtwvtymmih Wjxmmyvxzegdbtmfnyxe Cyvopca Oyofhrwkw I Tcjxcewcxffck Ii Mnckatggbff W Lfhhz Lodz Poland
Avpreen Sxr z ownb Poznan Poland
Aevgxtv Ucbsh Sbgujjhbj Lmhfra Dc Bzgozvw Bologna Italy
Umvfvsnplo Dgwiw Sahqf Dd Romh Li Siihbsys Rome Italy
Fnenehakt Pwpz Lv Iryminzxtvayq Bwkvoafqo Dmc Hzmmhuvf Utituohwjmvvc Lq Pxw Madrid Spain
Undwhsosqblurw Cbydixb Krcolqdvi Gdansk Poland
Hihomajf Vgmt dpavyrve Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not yet recruiting
15.12.2025
Czechia Czechia
Not yet recruiting
15.12.2025
France France
Recruiting
15.12.2025
Germany Germany
Not yet recruiting
15.12.2025
Hungary Hungary
Not yet recruiting
15.12.2025
Italy Italy
Not yet recruiting
15.12.2025
Poland Poland
Recruiting
15.12.2025
Spain Spain
Recruiting
15.12.2025

Trial locations

Investigated drugs:

Rilzabrutinib is an oral medication that works by targeting specific proteins in the body that are involved in the immune system’s response. It is being studied as a treatment for immune thrombocytopenia (ITP), a condition where the immune system mistakenly attacks and destroys platelets in the blood. This medication aims to help maintain healthy platelet levels in patients who haven’t responded well to their first treatment for ITP. It comes in tablet form that patients can take by mouth.

Immune Thrombocytopenia (ITP) – A blood disorder where the immune system mistakenly attacks and destroys platelets, which are blood cells needed for normal blood clotting. The condition causes lower than normal platelet counts in the blood, which can affect the body’s ability to control bleeding. ITP can develop suddenly or gradually, and it may occur after a viral infection or without any obvious trigger. The condition can affect both children and adults, though it often presents differently in each age group. People with ITP may experience symptoms such as easy bruising, small red or purple spots on the skin called petechiae, and prolonged bleeding from minor cuts.

Trial ID:
2025-522070-36-00
Protocol code:
LPS18573
NCT ID:
NCT07007962
Trial Phase:
Therapeutic confirmatory (Phase III)

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