Study on Long-Term Safety and Tolerability of Iptacopan for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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What is this study about?

This clinical trial is focused on studying the long-term safety and tolerability of a medication called iptacopan in patients with a rare blood disorder known as Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The medication being tested, iptacopan, is taken in the form of hard gelatin capsules and is designed to help manage this condition.

The purpose of the study is to evaluate how safe and well-tolerated iptacopan is when used over a long period. Participants in this study are individuals who have already completed previous phases of clinical trials with iptacopan. The study will monitor participants for any side effects or health changes while they continue taking the medication. This includes regular check-ups and assessments to ensure the medication is working as intended without causing harm.

Throughout the study, participants will be observed for any adverse events, which are unexpected health issues that may arise. The study will also track the stability of hemoglobin levels, which are important for carrying oxygen in the blood, and check for any need for blood transfusions. Additionally, the study will look for any occurrences of breakthrough hemolysis, which is when red blood cells break down too quickly, and any major vascular events, which are serious issues related to blood vessels. The study aims to provide valuable information on the long-term use of iptacopan for managing PNH.

1 enrollment

Participation begins after completing previous Phase II or Phase III studies with iptacopan.

Eligibility requires a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) and being clinically stable on iptacopan for at least 3 months.

Vaccinations against certain infections must be up to date.

2 treatment

Receive iptacopan in the form of hard gelatin capsules.

The medication is taken orally as prescribed by the study protocol.

The objective is to evaluate the long-term safety and tolerability of iptacopan.

3 monitoring

Regular safety evaluations are conducted, including monitoring for any adverse events.

Laboratory tests and vital signs are checked periodically.

The study aims to maintain hemoglobin levels and avoid blood transfusions.

4 follow-up

Yearly follow-up intervals assess the absence of blood transfusions and occurrences of breakthrough hemolysis.

Monitoring for Major Adverse Vascular Events (MAVE) is included in the follow-up.

5 completion

The study is estimated to end by February 7, 2028.

Final evaluations are conducted at the end of the study period.

Who Can Join the Study?

  • Participants must be 18 years or older.
  • Participants must have a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood condition.
  • Participants must have completed the treatment extension period of previous Phase II or any Phase III clinical studies with iptacopan, a medication being studied.
  • Participants must have received all necessary vaccinations against certain infections, including Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae. These are types of bacteria that can cause serious infections.
  • The study doctor must believe that the participant may benefit from continuing treatment with iptacopan and that the participant has been stable on this treatment for at least 3 months.

Who Cannot Join the Study?

  • Participants who have not completed the treatment extension period of the previous Phase II and any Phase III clinical studies with iptacopan.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Careggi Florence Italy
Oncopole Claudius Regaud Toulouse France
Technische Universitaet Dresden Dresden Germany
Centre Hospitalier Universitaire De Lille Lille France

Other Sites

Site Name City Country Status
Universitaetsklinikum Aachen AöR Aachen Germany
AORN San Giuseppe Moscati Avellino Avellino Italy
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Regione Del Veneto Azienda ULSS N 7 Pedemontana Bassano Del Grappa Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Clinic De Barcelona Barcelona Spain
Vilniaus universiteto ligonine Santaros klinikos VšĮ Vilnius Lithuania
Fakultni Nemocnice Brno Brno Czechia
Stichting Radboud University Medical Center Nijmegen The Netherlands
Universidade De Santiago De Compostela Santiago De Compostela Spain
ELBLANDKLINIKEN Stiftung & Co. KG Riesa Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Centre Hospitalier Universitaire De Nice Nice France
Azienda Sanitaria Territoriale Di Ascoli Piceno Ascoli Piceno Italy
University Hospital Ostrava Ostrava Czechia
Upairixexh Mknfmne Crpdpp Hfchhieuabmbrstak Hamburg Germany
Ufyrgkwcyawlmndcadqce Elgey Ajt Essen Germany
Ajvqgszpmy Padwuroa Hjzefiia Dd Prexa Paris France
Hkfcwwem Uwzivtjtcauye Dzlcsfao Donostia / San Sebastian Spain
Ugceettlgg Dblqg Srsjv Dy Rpdd Ls Sqnizben Rome Italy

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Czechia Czechia
Not recruiting
27.07.2021
France France
Not recruiting
27.07.2021
Germany Germany
Not recruiting
27.07.2021
Italy Italy
Not recruiting
27.07.2021
Lithuania Lithuania
Not recruiting
27.07.2021
Spain Spain
Not recruiting
27.07.2021
The Netherlands The Netherlands
Not recruiting
27.07.2021

Trial locations

Investigated drugs:

Iptacopan is a medication being studied for its long-term safety and tolerability in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This trial is for patients who have already completed earlier phases of studies with iptacopan. The goal is to understand how well patients can tolerate the medication over an extended period and to monitor any potential side effects.

Paroxysmal Nocturnal Hemoglobinuria (PNH) – This is a rare blood disorder characterized by the destruction of red blood cells, leading to symptoms such as fatigue, shortness of breath, and dark-colored urine. The disease occurs due to a mutation in the PIGA gene, which affects the surface proteins that protect red blood cells from the immune system. Over time, the destruction of red blood cells can lead to anemia and other complications. Patients may experience episodes of hemolysis, where red blood cells break down more rapidly, often at night. The condition can also increase the risk of blood clots and other vascular issues. PNH is a chronic condition that requires ongoing monitoring and management.

Trial ID:
2023-509843-28-00
Protocol code:
CLNP023C12001B
Trial Phase:
Therapeutic confirmatory (Phase III)

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