Immune ThrombocytopeniaPhase 3LUNA 4NCT07007962
Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
A Phase 3 study testing rilzabrutinib in people with Immune Thrombocytopenia, run by Sanofi at 36 sites in 9 countries.
Trial summary
This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- 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
- 60 participants
- Where
- 36 sitesin 9 countries
- Study dates
- 24 Oct 2025to 30 Dec 2028
- Phase
- Phase 3
- 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 or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history
- Participant received at least one course of first-line therapy and had a history of response while on treatment
- Participant has loss of response, relapse, or steroid dependency
Who cannot join
- Participants with Secondary ITP
- Participants with Evans syndrome or history of myelodysplastic syndrome
- Participants with history of lymphoma, leukemia, or any malignancy within the past 5 years except for non-melanoma skin malignancy.
- Participants with history of solid organ transplant
- Participants with history of coagulation or bleeding disorders other than ITP, including genetic conditions, other than ITP
- Participant received advanced therapy for ITP or was splenectomized
- Pregnancy or nursing 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
36 sites in 9 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
36 sites in 9 countries
United States12 sites, 12 recruiting
- Recruiting
New York Oncology Hematology-Investigational Site Number: 8400010
Albany, New York, 12208
- Recruiting
University of Michigan Health - Michigan Medicine - University Hospital-Investigational Site Number: 8400001
Ann Arbor, Michigan, 48109
- Recruiting
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
- Recruiting
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
- Recruiting
Community Health Partners (CHP) - Community Medical Oncology Specialists (University Oncology Associates)
Clovis, California, 93611
- Recruiting
Mass General Brigham Cancer Institute
Danvers, Massachusetts, 01923
- Recruiting
University of Southern California (USC)
Los Angeles, California, 90089
- Recruiting
Tulane University Health Sciences Center - Tulane Avenue-Investigational Site Number: 8400011
New Orleans, Louisiana, 70112
- Recruiting
Community Cancer Trials of Utah-Investigational Site Number: 8400002
Ogden, Utah, 84405
- Recruiting
Oregon Health and Sciences University
Portland, Oregon, 97239
- Recruiting
Mayo Clinic_Investigational Site Number: 8400009
Rochester, Minnesota, 55905
- Recruiting
Montefiore Medical Center-Investigational Site Number: 8400012
The Bronx, New York, 10467
Spain7 sites, 7 recruiting
- Recruiting
Investigational Site Number : 7240001
Barcelona, 8003
- Recruiting
Investigational Site Number : 7240002
Burgos, 9006
- Recruiting
Investigational Site Number : 7240007
Madrid, 28007
- Recruiting
Investigational Site Number : 7240008
Madrid, 28046
- Recruiting
Investigational Site Number : 7240005
Murcia, 30008
- Recruiting
Investigational Site Number : 7240003
San Cristóbal de La Laguna
- Recruiting
Investigational Site Number : 7240004
Seville, 41013
Italy5 sites, 5 recruiting
- Recruiting
Investigational Site Number : 3800002
Meldola
- Recruiting
Investigational Site Number : 3800004
Milan, 20162
- Recruiting
Investigational Site Number : 3800006
Naples, 80131
- Recruiting
Investigational Site Number : 3800007
Torino, 10126
- Recruiting
Investigational Site Number : 3800003
Vicenza, 36100
France4 sites, 4 recruiting
- Recruiting
Investigational Site Number: 2500003
Créteil, 94000
- Recruiting
Investigational Site Number : 2500001
Dijon, 21079
- Recruiting
Investigational Site Number : 2500002
Pessac, 33604
- Recruiting
Investigational Site Number : 2500004
Toulouse, 31059
Poland3 sites, 3 recruiting
- Recruiting
Investigational Site Number : 6160002
Gdansk, 80-219
- Recruiting
Investigational Site Number : 6160003
Skorzewo, 60-185
- Recruiting
Investigational Site Number : 6160004
Słupsk, 76-200
Hungary2 sites, 2 recruiting
- Recruiting
Investigational Site Number : 3480001
Budapest
- Recruiting
Investigational Site Number : 3480002
Kaposvár, 7400
Austria1 site, 1 recruiting
- Recruiting
Investigational Site Number : 0400001
Vienna, 1140
Czechia1 site, 1 recruiting
- Recruiting
Investigational Site Number : 2030002
Prague, 12800
Germany1 site, 1 recruiting
- Recruiting
Investigational Site Number : 2760004
Leipzig, 04103
About this study
Official title: A Multi-center, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.
- Conditions
- Immune Thrombocytopenia
- Being tested
- rilzabrutinib
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
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
| Study group | Role | What they receive |
|---|---|---|
| rilzabrutinib | Experimental | 400 mg BID Receives: Drug: rilzabrutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Durable platelet response
Defined as the percentage of participants able to achieve platelet counts ≥50 000/μL (or ≥30 000/μL and <50 000/μL and at least double from baseline) for ≥50% of 6 biweekly scheduled platelet measurements and at least 4 non-missing, biweekly visits during the last 12 weeks of the primary analysis period (PAP) in absence of rescue therapy
Measured: Until Week 28
Secondary outcomes (6)
Overall platelet response
Percentage of participants able to achieve 2 platelet counts at least 5 days apart of ≥50 000/μL (or ≥30 000/μL and <50 000/μL and at least double from baseline) without rescue therapy in the 4 weeks prior to the first elevated platelet count
Measured: Until Week 28
Duration of platelet response
Cumulative number and proportion of non-missing weeks with platelet counts ≥50 000/μL (or ≥30 000/μL and <50 000/μL and at least double from baseline) in absence of rescue therapy in the 4 weeks prior to the elevated platelet count in participants who achieve a response (single platelet count)
Measured: Until Week 28
Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28
Measured: Until Week 28
Percentage of participants able to discontinue or reduce CS dose by at least 50% or to <5 mg/day (prednisone equivalent) from baseline at the end of week 28
Measured: Until Week 28
Frequency and severity of treatment emergent adverse events (TEAEs, including serious adverse events [SAEs], bleeding TEAEs, adverse event of special interest [AESI] and adverse events leading to discontinuation)
Measured: Until Week 80
Percentage of participants with potential clinical significant abnormal (PCSA)
PCSA in physical examination, ECG, vital signs, and clinical laboratory test results: serum chemistry and hematology (except for platelet counts included in the primary efficacy endpoint)
Measured: Until Week 80
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2025
- Study start24 Oct 2025
- Primary completion5 Nov 2027 (estimated)
- Study completion30 Dec 2028 (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 Immune ThrombocytopeniaStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07007962
- Sponsor's protocol ID
- LPS18573
- Other IDs
- U1111-1320-4669
- Acronym
- LUNA 4
- Status last verified
- Jun 2026
- First submitted
- 20 May 2025
- Last updated
- 22 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
Mjali A, Audia S, Miyakawa Y, Ghanima W. Rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP): a Bruton tyrosine kinase inhibitor that can target multiple pathophysiological pathways. Expert Rev Hematol. 2026 Jul;19(7):685-696. doi: 10.1080/17474086.2026.2672113. Epub 2026 May 13.
PubMed 42106923
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
- ClinicalTrials.gov ID
- NCT07007962
- Acronym / sponsor ID
- LUNA 4 / LPS18573
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 24 Oct 2025 (actual)
- Primary completion
- 5 Nov 2027 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 6 Jun 2025 (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.
This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.
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: rilzabrutinib400 mg BID |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history
- Participant received at least one course of first-line therapy and had a history of response while on treatment
- Participant has loss of response, relapse, or steroid dependency
Key exclusion criteria (7 listed)
- Participants with Secondary ITP
- Participants with Evans syndrome or history of myelodysplastic syndrome
- Participants with history of lymphoma, leukemia, or any malignancy within the past 5 years except for non-melanoma skin malignancy.
- Participants with history of solid organ transplant
- Participants with history of coagulation or bleeding disorders other than ITP, including genetic conditions, other than ITP
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: SAR444671Pharmaceutical form:Tablet-Route of administration:Oral | Drug | rilzabrutinib |
Primary outcome
Durable platelet response
Time frame: Until Week 28
Defined as the percentage of participants able to achieve platelet counts ≥50 000/μL (or ≥30 000/μL and <50 000/μL and at least double from baseline) for ≥50% of 6 biweekly scheduled platelet measurements and at least 4 non-missing, biweekly visits during the last 12 weeks of the primary analysis period (PAP) in absence of rescue therapy
6 secondary outcomes
Overall platelet response
Time frame: Until Week 28
Duration of platelet response
Time frame: Until Week 28
Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28
Time frame: Until Week 28
Percentage of participants able to discontinue or reduce CS dose by at least 50% or to <5 mg/day (prednisone equivalent) from baseline at the end of week 28
Time frame: Until Week 28
Frequency and severity of treatment emergent adverse events (TEAEs, including serious adverse events [SAEs], bleeding TEAEs, adverse event of special interest [AESI] and adverse events leading to discontinuation)
Time frame: Until Week 80
Percentage of participants with potential clinical significant abnormal (PCSA)
Time frame: Until Week 80
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 22 Jun 2026.
- Derived from this study: PubMed 42106923Mjali A, Audia S, Miyakawa Y, Ghanima W. Rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP): a Bruton tyrosine kinase inhibitor that can target multiple pathophysiological pathways. Expert Rev Hematol. 2026 Jul;19(7):685-696. doi: 10.1080/17474086.2026.2672113. Epub 2026 May 13.
- LPS18573 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.
Trial Transparency email recommended (Toll free for US & Canada) contact
36 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing rilzabrutinib in people with Immune Thrombocytopenia, run by Sanofi at 36 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 3 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 22 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 36 sites in United States, Spain, Italy, France, Poland 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Contact Trial Transparency email recommended (Toll free for US & Canada) (800-633-1610 ext. option 6, contact-us@sanofi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Durable platelet response (time frame: Until Week 28). 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 NCT07007962, 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 22 Jun 2026.