Novartis withdraws planned phase 2 retreatment study of ianalumab in ITP and wAIHA

Novartis Pharmaceuticals registered a study titled "A Phase 2 Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab."¹ The trial, listed under NCT07039422 and acronym VAY RE-HIT, has an overall status of withdrawn, with the sponsor citing evolving scientific and feasibility considerations that could adversely affect recruitment, reducing the likelihood of generating a sufficiently robust dataset to support reliable efficacy and safety conclusions.¹

The study's record lists an estimated start of 2026-06-26 and estimated primary and study completion of 2030-08-30, with the record first posted 2025-06-26 and last updated 2026-10-15. Enrollment was recorded at "Enrollment: 0 participants (actual)."¹

The study was designed to examine whether a second course of ianalumab could be effective and safe after treatment failure in the pivotal ITP trials (CVAY736I12301, CVAY736Q12301) and after loss of durable response in the pivotal wAIHA trial (CVAY736O12301).¹ The planned design called for a multicenter, phase 2 exploratory study in which all participants would receive ianalumab at the dose from which they had previously derived benefit.¹

Planned primary outcomes included treatment failure free status for ITP participants by 12 months after the start of a second course of ianalumab at the assigned dose¹ and, for wAIHA, durable response defined as hemoglobin of at least 10 g/dL with at least a 2 g/dL increase from baseline sustained for at least 8 consecutive weeks between week 9 and week 25, without rescue or prohibited treatment before that point.¹