Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
NCT04820530 · APPOINT-PNH Phase 3Completed
- Lead sponsor
- Novartis Pharmaceuticals (Industry)
- Enrollment
- 40 (actual)
- Start date
- 19 Jul 2021
- Primary completion
- 2 Nov 2022 (actual)
- Study completion
- 18 Apr 2023
- Last update posted
- 9 Oct 2024
Design
- Study type
- Interventional
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None
- Arms
- 1
Interventions
- Iptacopan (LNP023) Drug
Conditions and primary outcome
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary outcome
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 (Baseline, hemoglobin between Day 126 and Day 168 and absence of transfusions between Day 14 and Day 168)
Posted results on ClinicalTrials.gov
Source: ClinicalTrials.gov results for NCT04820530 (opens in a new tab)· Licence: public-domain
First posted · Last update posted · Imported from ClinicalTrials.gov download 2026-10-25
Primary outcomes with a statistical analysis
No primary outcome with a statistical analysis is posted for this study.
Serious adverse events
Time frame: Adverse events of LNP023 group were reported from first dose of study treatment until the end of study treatment plus up to 30 days, up to a maximum duration of 48 weeks
| Group | Affected | At risk |
|---|---|---|
| LNP023 200mg b.i.d. | 8 | 40 |
Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.
Results from company and journal sources
No editor-confirmed results are on record for this study.
Publications
3 on PubMedPatient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan.
The patient · 2025 Nov
PubMed 40694294 (opens in a new tab)DOI 10.1007/s40271-025-00755-5 (opens in a new tab)
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.
The Lancet. Haematology · 2025 Jun
PubMed 40447351 (opens in a new tab)DOI 10.1016/S2352-3026(25)00081-X (opens in a new tab)
Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.
The New England journal of medicine · 2024 Mar 14
PubMed 38477987 (opens in a new tab)DOI 10.1056/NEJMoa2308695 (opens in a new tab)
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.
CT-VCA trial quality
About CT-VCALoading CT-VCA scores…
Data integrity
Compare what the registry, company releases, FDA documents and publications say about this study.
Drugs on HealthTrade
Source
- NCT04820530 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.
Nothing on this page is medical or investment advice.