A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids

Summary

The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.

Timeline

Start

2023-02-02

Primary completion

2025-06-19

Completion

2028-04-08

Outcome

Met primary endpoint

paper The time to treatment failure was significantly longer with ianalumab plus eltrombopag than with placebo plus eltrombopag; PMID 41363800 ↗

paper the estimated hazard ratio for treatment failure (ianalumab vs. placebo) was 0.55 (P = 0.04) in the 9-mg group PMID 41363800 ↗

release “In the Phase III VAYHIT2 trial, ianalumab plus eltrombopag significantly extended the time to treatment failure” novartis.com ↗

release “Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45%, based on the primary endpoint of time to treatment failure (TTF)” novartis.com ↗

Publications

• Cuker A, Stauch T, Cooper N, Al-Samkari H, Michel M, Ghanima W, Urban P, Fronczek J, Foster M, Weill M, Zhang L, Hou M, Zander T, Sharif A, Sun J, Nath UK, Schutgens R, Rossi E, Deleu L, Cervinek L, Yoon JH, Chang H, Ruchutrakool T, Iino M, Goto T, Zaja F; VAYHIT2 Investigators. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. N Engl J Med. 2026 Apr 16;394(15):1503-1513. doi: 10.1056/NEJMoa2515168. Epub 2025 Dec 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ianalumab	Monoclonal antibody	3 mg/kg	Intravenous
Subject	Ianalumab	Monoclonal antibody	9 mg/kg	Intravenous
Background	Eltrombopag	Small molecule	—	Oral

Indications