Actively Recruiting
A Phase 2 Open-label Study to Evaluate the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least One Treatment (VAY2EXPLORE)
Led by Novartis Pharmaceuticals · Updated on 2026-05-08
164
Participants Needed
2
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the tolerability of ianalumab combined with an investigator's choice thrombopoietin receptor agonist (IC TPO-RA) in adults diagnosed with primary immune thrombocytopenia (ITP) who have received between one and four prior treatments. The study also includes an exploratory group of participants with primary Evans syndrome (ES) who are considered suitable for IC TPO-RA therapy. This phase 2 open-label study aims to assess how well participants tolerate this combination treatment and monitor its effects over time. The study involves a 28-day screening period followed by a 16-week treatment phase where participants receive ianalumab at a dose of 9 mg per kilogram via infusion every four weeks, alongside their chosen IC TPO-RA administered per approved guidelines. After treatment, there is a tapering period for the IC TPO-RA lasting 16 weeks, during which participants are closely monitored. Following these phases, participants enter a long-term safety follow-up lasting 60 weeks (about 15 months) to observe ongoing effects and safety. Participants will undergo regular assessments including platelet counts at scheduled visits throughout the study. Researchers will track tolerability to ianalumab up to week 16, monitor adverse events over an average of four years, and evaluate platelet count changes and success in tapering IC TPO-RA. Safety and treatment effects will be carefully followed during the long-term period. Total involvement spans the screening, treatment, tapering, and extended follow-up phases, ensuring thorough monitoring of participant health and treatment response.
CONDITIONS
Brief Title
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent prior to participation
- Adults aged 18 years and older at consent
- For ITP cohort: Confirmed primary ITP diagnosis with prior response to corticosteroids or IVIG but not sustained
- For ITP cohort: At least one prior treatment for ITP
- For ITP cohort: Platelet count below 100 G/L and receiving a stable dose of TPO-RA for at least 14 days before ianalumab
- For ES cohort: Clinical diagnosis of primary Evans syndrome with active thrombocytopenia (platelets < 100 G/L) and warm autoimmune hemolytic anemia
- For ES cohort: Inadequate response or relapse after corticosteroid therapy
- For ES cohort: Positive direct antiglobulin test (IgG+, with or without C3+) and evidence of hemolysis
- For ES cohort: Stable supportive care for warm autoimmune hemolytic anemia for at least 4 weeks prior to enrollment
You will not qualify if you...
- Treatment with TPO-RA for more than 6 months
- Current life-threatening bleeding related to thrombocytopenia
- Splenectomy within 6 months before starting ianalumab
- Laboratory abnormalities including neutrophils below 1000/mm3, high serum creatinine, elevated AST or ALT, low IgG, and for ITP cohort, low hemoglobin or high bilirubin
- Significant or decompensated liver disease
- Treatment with B-cell depleting therapy or anti-B cell activating factor within 12 weeks prior to ianalumab
- Refractory to rituximab treatment
- History of hypersensitivity to study drugs or similar classes
- Known primary or secondary immunodeficiency, or positive HIV test
- Exposure to more than 4 prior ITP treatments
- For ITP cohort: Secondary thrombocytopenia diagnosis
- Use of immunosuppressants other than corticosteroids or rituximab in ITP cohort
- For ES cohort: Secondary Evans syndrome diagnosis
- Life-threatening hemolysis in ES cohort
- Autoimmune hemolytic anemia other than warm type in ES cohort
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 28 days
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 16 weeks
Participants receive ianalumab infusions every 4 weeks combined with investigator's choice thrombopoietin receptor agonist (TPO-RA) therapy.
4 infusions every 4 weeks plus additional visits for TPO-RA administration as per prescribing information
Duration - 16 weeks
Participants are monitored while tapering off the thrombopoietin receptor agonist (TPO-RA).
Visits scheduled throughout tapering period for monitoring
Duration - 60 weeks
Participants are followed for safety monitoring for up to 60 weeks after tapering.
Periodic visits during follow-up phase
Trial Site Locations
Total: 2 locations
1
DH Cancer Research Center LLC
Margate, Florida, United States, 33063
Actively Recruiting
2
Hope And Healing Care
Hinsdale, Illinois, United States, 60521
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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