Immune Thrombocytopenic Purpura (ITP) is a blood disorder where the immune system attacks platelets. This causes low platelet counts and bleeding risks.
Several promising clinical trials are underway as of March 2025. These trials test new treatments that may improve outcomes for patients.
FA
by FNU Ashish
Understanding ITP
Blood Disorder
ITP causes the immune system to destroy platelets needed for blood clotting.
Symptoms
Easy bruising, small purple dots on skin (petechiae), and prolonged bleeding.
Diagnosis
Low platelet counts below 30,000/μL indicate the condition.
Clinical Trial Phases
Phase I
Tests safety and identifies side effects in small groups.
Phase II
Evaluates effectiveness and continues safety monitoring.
Phase III
Large-scale testing for efficacy and side effects before approval.
VAYHIT1 Trial
Participants
Adults ≥ 18 years with newly diagnosed primary ITP within 3 months.
Treatment
Ianalumab (high/low dose) or placebo, with corticosteroids.
Goal
Maintain platelet count ≥ 30G/L without rescue therapy.
Status
Currently recruiting patients as of March 2025.
VAYHIT2 and HMPL-523 Trials
VAYHIT2 Trial
Tests ianalumab with eltrombopag for second-line therapy.
Aims to improve long-term outcomes for patients who failed initial treatment.
Currently ongoing but not recruiting new patients.
HUTCHMED Trial
Evaluates HMPL-523, a selective Syk inhibitor.
For adults diagnosed with ITP ≥ 3 months ago.
Recently started, likely still recruiting participants.
Pediatric and Adolescent Trials
1
Eltrombopag Trial
Compares eltrombopag with standard front-line therapy in children ages 1-18.
2
Rilzabrutinib Study
Tests rilzabrutinib for persistent/chronic ITP in adults and adolescents.
3
Multicenter Approach
Collaborative effort across institutions like Baylor College of Medicine.
Trial Status Summary
Recruiting
VAYHIT1 trial is actively enrolling new patients.
Ongoing
VAYHIT2 and HUTCHMED trials are in progress.
Closed to New Patients
Pediatric eltrombopag and rilzabrutinib trials not accepting new participants.
Future Developments
LUNA 3 study met endpoints for rilzabrutinib in April 2024.
Patient Resources
ClinicalTrials.gov
Visit ClinicalTrials.gov for the most up-to-date listings of ITP trials.