Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

Phase 2 Terminated 19 enrolled Novartis Pharmaceuticals

NaSingle-groupOpen-labelTreatment

Summary

The main purpose of this study was to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.

Timeline

Start: 2021-12-21
Primary completion: 2023-09-20
Completion: 2024-05-17

Outcome

Mixed primary results

Stopped (Business): “Business decision”

paper five (50%) patients achieved the primary endpoint. PMID 41366634 ↗

paper Conversely, no patients with ITP met the primary endpoint. PMID 41366634 ↗

Publications

Roth A, Barcellini W, Ademokun C, Jang J, Lozano ML, Ferreiras DV, Pascual-Izquierdo C, Chitnis S, Matviykiv S, Vitaliti A, Chen C, Katsanou V, Chawla R, Al-Samkari H. Iptacopan for Immune Thrombocytopenia and Cold Agglutinin Disease: A Global Phase 2 Basket Clinical Trial. Am J Hematol. 2026 Feb;101(2):242-254. doi: 10.1002/ajh.70147. Epub 2025 Dec 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Iptacopan	Small molecule	200 mg	Oral