Rilzabrutinib for Immune Thrombocytopenia

Not currently recruiting at 71 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment, Rilzabrutinib (also known as PRN1008), for individuals with immune thrombocytopenia (ITP). ITP is a disorder where the immune system mistakenly destroys platelets, which help blood clot. The trial aims to determine if Rilzabrutinib can safely increase platelet counts in those whose ITP has not improved with other treatments. Participants should have ITP and low platelet counts despite trying other therapies. If successful, this could offer new hope for those struggling with this condition. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to potential advancements in ITP treatment.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that rilzabrutinib is generally safe for people with immune thrombocytopenia (ITP). Studies found that while some side effects are common, they are usually mild. For instance, about 23% of patients taking rilzabrutinib experienced diarrhea, compared to 4% of those taking a placebo (a non-active treatment used for comparison). Other side effects, such as nausea, headache, and stomach pain, were also slightly more common in the rilzabrutinib group than in the placebo group.

Patients who used rilzabrutinib after other ITP treatments failed saw an improvement in their platelet levels. This suggests that the treatment can be effective for those who need it. Overall, while some side effects occur, most patients have tolerated the treatment well. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Rilzabrutinib is unique because it targets Bruton’s tyrosine kinase (BTK), offering a fresh approach to treating immune thrombocytopenia (ITP). While standard treatments like corticosteroids, immunoglobulins, and splenectomy focus on suppressing the immune system or altering platelet destruction, rilzabrutinib directly interferes with the signaling pathways that lead to platelet destruction. Researchers are excited about its potential to provide a more targeted and possibly safer treatment option, with fewer side effects compared to traditional therapies. Plus, its oral administration is convenient, making it easier for patients to incorporate into their daily routines.

What evidence suggests that Rilzabrutinib might be an effective treatment for immune thrombocytopenia?

Research has shown that rilzabrutinib, which participants in this trial will receive, may help treat immune thrombocytopenia (ITP). In one study, 64% of patients taking rilzabrutinib experienced a significant increase in their platelet counts, compared to only 32% of those taking a placebo. As a result, more patients reached safer platelet levels. Rilzabrutinib also worked quickly and had lasting effects, reducing the need for additional medication. These findings suggest that rilzabrutinib could be a good option for people with ITP who haven't had success with other treatments. ^{1 2 3 4 6}

Who Is on the Research Team?

Olga Bandman, MD

Principal Investigator

Principia Biopharma

Are You a Good Fit for This Trial?

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 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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

Citations

1. ashpublications.org ashpublications.org/blood/article/145/24/2914/536104/Safety-and-efficacy-of-rilzabrutinib-vs-placebo-in

Safety and efficacy of rilzabrutinib vs placebo in adults with ... Rilzabrutinib in patients who failed multiple previous ITP therapies showed rapid and durable platelet response, reduced rescue medication and bleeding, ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40090011/

Safety and efficacy of rilzabrutinib vs placebo in adults with ... Overall (N = 202), 85 (64%) rilzabrutinib and 22 (32%) placebo patients achieved platelet response (≥50 × 109/L or 30 × 109/L to <50 × 109/L and ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39844469/

Efficacy and Safety Results With Rilzabrutinib, an Oral ... Rilzabrutinib continues to demonstrate durable platelet responses with favorable safety profile in previously treated ITP patients.

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa2110297

Rilzabrutinib, an Oral BTK Inhibitor, in Immune ... Overall, treatment with oral rilzabrutinib led to rapid and durable clinical activity in 40% of these patients with immune thrombocytopenia, who ...

5. sanofi.com sanofi.com/en/media-room/press-releases/2024/2024-12-07-16-30-00-2993342

Press Release: ASH: rilzabrutinib demonstrated significant ... ASH: rilzabrutinib demonstrated significant patient benefit in the first positive phase 3 study of a BTK inhibitor in ITP.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04562766

NCT04562766 | Study to Evaluate Rilzabrutinib in Adults ... This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no ...