Rilzabrutinib for Immune Thrombocytopenia

Recruiting at 60 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Principia Biopharma, a Sanofi Company

Disqualifiers: Pregnancy, Drug abuse, Organ transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

81 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count \<30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study has been completed. Part B treatment dose is 400 mg twice daily.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Rilzabrutinib for treating Immune Thrombocytopenia?

Research shows that Rilzabrutinib, a drug that blocks a specific protein involved in immune responses, can help increase platelet counts in people with Immune Thrombocytopenia by reducing the destruction of platelets and the production of harmful antibodies. In a study, 40% of patients showed improved platelet levels without needing additional medication, and the drug was generally well-tolerated. ^{1 2 3 4 5}

Is Rilzabrutinib safe for humans?

Rilzabrutinib has been studied in patients with immune thrombocytopenia and showed a well-tolerated safety profile with only mild, temporary side effects. ^{1 2 3 6 7}

How is the drug Rilzabrutinib different from other treatments for immune thrombocytopenia?

Rilzabrutinib is unique because it is an oral drug that works by inhibiting Bruton's tyrosine kinase, which helps reduce the destruction of platelets and the production of harmful antibodies. This dual mechanism of action sets it apart from other treatments that typically focus on either reducing platelet destruction or increasing platelet production. ^{1 2 3 6 8}

Research Team

Olga Bandman, MD

Principal Investigator

Principia Biopharma

Eligibility Criteria

Adults aged 18 to 80 with immune-related ITP, a condition where the immune system destroys platelets, are eligible for this trial. Participants must have a low platelet count and not respond well to other treatments. Those with drug or alcohol abuse, organ transplants, HIV, hepatitis B/C, or who are pregnant/breastfeeding cannot join.

Inclusion Criteria

I am between 18 and 80 years old.

I have immune-related ITP.

Exclusion Criteria

You are currently misusing drugs or alcohol.

You have tested positive for HIV, hepatitis B, or hepatitis C during screening.

I have had a solid organ transplant.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive PRN1008 400mg BID for up to 24 weeks; includes safety and dose evaluation

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term extension

Participants who respond to PRN1008 may enter a long-term extension

Treatment Details

Interventions

• PRN1008

Trial Overview The study is testing Rilzabrutinib (PRN1008), specifically at a dose of 400 mg twice daily for adults with refractory or relapsed ITP after completing an initial dose-finding phase. The goal is to see if it can safely increase platelet counts in these patients.

Participant Groups

Group I: Rilzabrutinib (PRN1008) Daily Experimental Treatment 1 Intervention

Part A approximately 60 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension. Part B approximately 25 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension

Who Is Running the Clinical Trial?

Principia Biopharma, a Sanofi Company

Lead Sponsor

Trials

10

Recruited

670+

Findings from Research

In a phase 1-2 clinical trial involving 60 patients with immune thrombocytopenia, rilzabrutinib demonstrated a platelet response in 40% of participants, indicating its efficacy in increasing platelet counts through dual mechanisms of action.

The treatment was well-tolerated, with only low-level, transient side effects and no serious bleeding or thrombotic events, suggesting a favorable safety profile for further testing at the identified effective dose of 400 mg twice daily.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. Kuter, DJ., Efraim, M., Mayer, J., et al.[2022]

Fostamatinib is an oral spleen tyrosine kinase inhibitor that effectively increases platelet counts in adults with immune thrombocytopenia who did not respond adequately to previous treatments, as shown in two Phase III trials.

The treatment is generally well tolerated, with lower rates of severe bleeding-related side effects, although common adverse events include diarrhea, nausea, and liver function test elevations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of immune thrombocytopenia in adults. Moore, DC., Gebru, T., Muslimani, A.[2021]

In a 52-week study involving 234 adults with immune thrombocytopenia, romiplostim significantly increased the rate of platelet response compared to standard care, with a response rate 2.3 times higher (P<0.001).

Patients receiving romiplostim experienced fewer treatment failures and required splenectomy less often (11% vs. 30% for standard care, P<0.001), along with lower rates of bleeding events and improved quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Romiplostim or standard of care in patients with immune thrombocytopenia. Kuter, DJ., Rummel, M., Boccia, R., et al.[2016]

References

1. United States pubmed.ncbi.nlm.nih.gov

Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. [2023]

3. Italy pubmed.ncbi.nlm.nih.gov

Comparison of up-front treatments for newly diagnosed immune thrombocytopenia -a systematic review and network meta-analysis. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Treatment patterns in adults with immune thrombocytopenia before, during and after use of thrombopoietin receptor agonists: a longitudinal prescription database study from Germany. [2022]

5. Brazil pubmed.ncbi.nlm.nih.gov

Safety and efficacy of romiplostim in children and adolescents with Immune thrombocytopenia: A systematic review. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Fostamatinib for the treatment of immune thrombocytopenia in adults. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Romiplostim or standard of care in patients with immune thrombocytopenia. [2016]

8. England pubmed.ncbi.nlm.nih.gov

Rituximab therapy in adult patients with relapsed or refractory immune thrombocytopenic purpura: long-term follow-up results. [2015]