Phase 3
Completed N=40
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Source: ClinicalTrials.gov NCT04820530 ↗
Enrolled (actual)
40
Serious AEs
20.0%
Results posted
Nov 2023
Primary outcomePrimary: 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 — 92.2 Percentage of responders
◆ Published Evidence
Highly cited
143citations · ~72 / year
Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.
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.
Linked Publications (3)
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Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.
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Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors.
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Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
92.2 | — |
| SECONDARY Marginal Proportion (Expressed as Percentage) With Sustained Hemoglobin Levels of ≥ 12 g/dL in the Absence of Red Blood Cell Transfusions |
62.8 | — |
| SECONDARY Marginal Proportion (Expressed as Percentage) of Participants Who Remain Free From Transfusions |
97.6 | — |
| SECONDARY Change From Baseline in Hemoglobin Levels in the Core Treatment Period |
4.28 | — |
| SECONDARY Percent Change From Baseline in LDH |
-83.55 | — |
| SECONDARY Adjusted Annualized Clinical BTH Rate in the Core Treatment Period |
0.00 | — |
| SECONDARY Change From Baseline in Absolute Reticulocyte Count |
-82.48 | — |
| SECONDARY Change From Baseline in FACIT-Fatigue Score |
10.4 | — |
| SECONDARY Adjusted Annualized Major Adverse Vascular Events Rate in the Core Treatment Period |
0.00 | — |
Eligibility Criteria
Inclusion Criteria
- Male and female participants ≥ 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with RBCs and WBCs clone size ≥ 10%
- Mean hemoglobin level 1.5 x Upper Limit of Normal (ULN)
- Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment
- If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given
Exclusion Criteria
- Prior treatment with a complement inhibitor, including anti-C5 antibody
- Known or suspected hereditary complement deficiency
- History of hematopoietic stem cell transplantation
- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L).
- Active systemic bacterial, viral (incl. COVID-19)or fungal infection within 14 days prior to study drug administration.
- History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., NYHA class IV heart failure), severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.
Data sourced from ClinicalTrials.gov (NCT04820530) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.