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

Sponsor
Novartis Pharmaceuticals
Study ID
NCT04820530
Phase
PHASE3
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Jul 19, 2021
Status verified
Oct 2024
Primary completion
Nov 2, 2022
Completion
Apr 18, 2023

Study Design

Enrollment
40 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: LNP023
    Participants receive LNP023 at a dose of 200 mg orally b.i.d

Primary Outcome Measure

Marginal Proportion (Expressed as Percentage) of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 2 g/dL in the Absence of Red Blood Cell Transfusions [ Time Frame: Baseline, hemoglobin between Day 126 and Day 168 and absence of transfusions between Day 14 and Day 168 ]

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