Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Part of paid clinical trials in Duarte, California.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT04558918
Phase
PHASE3
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

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

Interventions

  • LNP023 — DRUG
    Taken Orally b.i.d. Dosage Supplied: 200 mg Dosage form: Hard gelatin capsule Route of Administration: Oral
  • Eculizumab — DRUG
    Administered as intravenous infusion every 2 weeks as per the stable regimen, the maintenance dose is a fixed dose. Dosage Supplied: 300 mg/30mL Dosage form: Concentrate solution for infusion
  • Ravulizumab — DRUG
    Administered as intravenous infusion every 8 weeks, the maintenance dose is based on body weight. Dosage Supplied: 300 mg/30mL Dosage form: Concentrate solution for infusion

Study Details

This study was a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this Phase 3 study in PNH patients presenting with residual anemia despite treatment with anti-C5 antibody, was to determine whether iptacopan is efficacious and safe for the treatment of PNH through demonstration of superiority of iptacopan compared to anti-C5 antibody treatment.

Key Dates

Start date
Jan 25, 2021
Status verified
Oct 2024
Primary completion
Sep 26, 2022
Completion
Mar 6, 2023

Study Design

Enrollment
97 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: LNP023 200mg b.i.d.
    Iptacopan 200mg b.i.d. hard gelatin capsule. After 24 weeks of LNP023 200mg b.i.d. treatment in the Randomized Treatment Period, participants had the option to enter the Extension Treatment Period to receive an additional 24 weeks of LNP023 200mg b.i.d.
  • Active Comparator: Anti-C5 antibody
    In the Randomized Treatment Period patients randomized to receive Anti-C5 antibody continued with the same stable regimen of Anti-C5 antibody therapy as they had received prior to randomization. For eculizumab (administered as intravenous infusion every 2 weeks), the maintenance dose was a fixed dose, whereas for ravulizumab (administered as intravenous infusion every 8 weeks), the maintenance dose was based on body weight. After 24 weeks of Anti-C5 antibody treatment in the Randomized Treatment Period, participants had the option to enter the Extension Treatment Period to receive 24 weeks of LNP023 200mg b.i.d.

Primary Outcome Measure

Marginal Proportion (Expressed as Percentages) 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 ]

Locations (5)

FacilityCityStateZIPSite coordinators
City Of Hope National Med Center City of Hope Medical CenterDuarteCalifornia91010-
Univ Cali Irvine ALS NeuromuscularOrangeCalifornia92868-
Augusta University Georgia Patient TreatmentAugustaGeorgia30912-
Levine Cancer Insitute Carolinas Healthcare SystemCharlotteNorth Carolina28204-
Cleveland Clinic Foundation Dept.ofTaussigCancer CenterClevelandOhio44195-

Find similar trials in Duarte, CA

By research site
City Of Hope National Med Center City of Hope Medical Center· Duarte, CAUniv Cali Irvine ALS Neuromuscular· Orange, CAAugusta University Georgia Patient Treatment· Augusta, GALevine Cancer Insitute Carolinas Healthcare System· Charlotte, NCCleveland Clinic Foundation Dept.ofTaussigCancer Center· Cleveland, OH

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