NCT05086744

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

An Open-label, Multi-center, Phase 2 Basket Study to Assess Efficacy, Safety and Pharmacokinetics of Iptacopan (LNP023) in Participants With Autoimmune Benign Hematological Disorders

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 120 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This was an open-label, single-arm (within each cohort), multi-center, non-confirmatory basket study to assess the efficacy, safety and pharmacokinetics of iptacopan in participants with autoimmune benign hematological disorders. The study was set up as a basket study to allow inclusion of new cohorts (indications). The study included 2 cohorts: primary immune thrombocytopenia (ITP) and primary cold agglutinin disease (CAD). Participants in Cohort 1 (ITP) were stratified in two groups according to high/low complement activation (i.e., based on sC5b-9 level at screening). No additional cohorts were added during the study. The study consisted of a screening period, a 12-week treatment period (Part A), a washout (for responders)/follow-up (for non-responders) period after Part A, and, for responders only, an additional treatment extension period for up to 24 months (Part B). Non-responders who had signs of clinical benefit according to the Investigator's assessment could also continue treatment with iptacopan in Part B. The washout period prior to the start of part B lasted up to 4 weeks.

Conditions

• Immune Thrombocytopenia (ITP)
• Cold Agglutinin Disease (CAD)

Interventions

Type	Name	Description
DRUG	Iptacopan	Iptacopan 200 mg BID given orally (capsule)

Timeline

• Start date: 2021-12-21
• Primary completion: 2023-09-20
• Completion: 2024-05-17
• First posted: 2021-10-21
• Last updated: 2025-10-16
• Results posted: 2025-03-19

Locations

8 sites across 6 countries: United States, Germany, Italy, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study