NCT05648968

A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia

A Phase 3, Randomized, Double-blind, Study to Assess Efficacy and Safety of Ianalumab (VAY736) Versus Placebo in Warm Autoimmune Hemolytic Anemia (wAIHA) Patients Who Failed at Least One Line of Treatment

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 90 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate efficacy and safety of ianalumab compared to placebo in patients with warm autoimmune hemolytic anemia, who failed at least one line of treatment.

Detailed description

The primary objective is to demonstrate that either dose of ianalumab induces a durable hemoglobin response compared to placebo in patients with wAIHA. The key secondary objective is to demonstrate that either dose of ianalumab maintains a durable hemoglobin response that is sustained beyond end of the treatment period, compared to placebo. Participants are randomized to two different doses of ianalumab or placebo. Participants who were assigned to placebo arm and not responding to treatment may be treated with open label ianalumab using the higher dose. The investigational treatment will be supplied in a double-blinded manner. For the open label period, ianalumab will be provided in an open label manner. In addition to the randomized treatment (ianalumab or placebo), specific supportive care medication as defined in the protocol is allowed. If clinically indicated (e.g., to ensure patient safety), the treating physician may also administer rescue medication. The study consists of the treatment period, efficacy and safety follow-up periods. The visit frequency will be every other week during the treatment and primary endpoint follow up period; for safety monitoring monthly during the first 20 weeks after last dose and afterwards quarterly up to 2 years from the last dose. For participants in durable response, additional visits for efficacy will occur monthly during the first 2 years after the last dose, and afterwards quarterly until loss of response or end of study, latest until up to 39 months post randomization of the last participant.

Conditions

• Warm Autoimmune Hemolytic Anemia (wAIHA)

Interventions

Type	Name	Description
BIOLOGICAL	Ianalumab	i.v. infusion, prepared from concentrate solution
DRUG	Placebo	i.v. infusion, prepared from matching placebo

Timeline

• Start date: 2022-12-30
• Primary completion: 2026-02-20
• Completion: 2028-12-19
• First posted: 2022-12-13
• Last updated: 2026-03-19

Locations

53 sites across 16 countries: United States, Argentina, Australia, China, France, Germany, Hungary, India, Israel, Italy, Japan, Malaysia, Singapore, Spain, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study