NCT04820530

Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this Phase 3 study was to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) patients who were naïve to complement inhibitor therapy.

Detailed description

This study was a multicenter, single-arm, open-label trial which was comprised of 8 weeks screening period, 24-week core treatment period and 24-week extension treatment period. Eligible PNH patients with hemolysis (LDH \> 1.5 ULN) and anemia (hemoglobin \<10 g/dL), who were naive to complement inhibitor therapy, including anti-C5 antibody treatment, received iptacopan monotherapy at a dose 200 mg orally b.i.d.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Type	Name	Description
DRUG	Iptacopan (LNP023)	Taken orally b.i.d. Dosage supplied: 200mg Dosage form: Hard gelatin capsule Route of Administration: oral

Timeline

• Start date: 2021-07-19
• Primary completion: 2022-11-02
• Completion: 2023-04-18
• First posted: 2021-03-29
• Last updated: 2024-10-09
• Results posted: 2023-11-15

Locations

12 sites across 8 countries: China, France, Germany, Italy, Malaysia, Singapore, South Korea, United Kingdom

Regulatory

• FDA-regulated drug study