Immune Thrombocytopenia
Conditions
Brief 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. 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.
Interventions
DRUGrilzabrutinib
Pharmaceutical form:Tablet-Route of administration:Oral
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * 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
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Durable platelet response | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Overall platelet response | Until Week 28 | 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 |
| Duration of platelet response | Until Week 28 | 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) |
| Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28 | 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 | 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) | Until Week 80 | — |
| Percentage of participants with potential clinical significant abnormal (PCSA) | Until Week 80 | 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) |
Countries
Austria, Czechia, France, Germany, Hungary, Italy, Poland, Spain, United States
Contacts
CONTACTTrial Transparency email recommended (Toll free for US & Canada)
Outcome results
None listed