Active not recruitingPHASE3INTERVENTIONAL

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

This ongoing research study is designed for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. It focuses on understanding the long-term safety and how well a medicine called iptacopan works over time. This particular study is for people who have already been taking iptacopan in earlier studies (Phase 2 or Phase 3 trials) and have found it beneficial. The main goal is to continue providing iptacopan to these patients while carefully monitoring their health. This helps gather more information about its effects over many years. It's a key step to ensure that people who benefit from iptacopan can keep receiving it safely.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

208

Start

27 Jul 2021

Estimated completion

18 Oct 2027

Paroxysmal Nocturnal Hemoglobinuria

What is this study about?

This study is about a medicine called iptacopan, designed for people living with a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. PNH is a disorder where red blood cells are destroyed too early, which can lead to anaemia, tiredness, and a risk of blood clots. Iptacopan is being studied as a potential treatment to help manage these symptoms and improve the lives of people with PNH.

This specific study is a continuation for patients who have already participated in previous research studies (called Phase 2 or Phase 3 trials) where they received iptacopan. The researchers want to keep a close eye on these patients over a longer period. This will help them understand how safe iptacopan is when taken for many years and how effective it remains in controlling PNH symptoms. It also ensures that patients who have already found iptacopan helpful can continue to receive this treatment.

By carefully tracking the health of these patients, doctors can learn more about the long-term effects of iptacopan. This information is crucial for future decisions about the medicine and helps ensure that patients with PNH have access to treatments that are both safe and effective over their lifetime.

Key takeaways

This study is for adults with PNH who have already used iptacopan.
It aims to check the long-term safety and effectiveness of iptacopan.
Participation means continuing iptacopan with regular health checks.
It allows patients who benefit from iptacopan to keep receiving it.
You can stop participating at any time if you wish.

Who may be eligible?

To be considered for this study, you must be an adult aged 18 or older and have a diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH). A key requirement is that you must have already completed treatment in one of the previous iptacopan studies (either Phase 2 or Phase 3 trials) and were still taking the medication when that study ended, meaning you didn't stop or reduce the dose on your own.

Additionally, your doctor must believe that you are benefiting from iptacopan and have been stable on the treatment for at least three months. You also need to have received certain vaccinations against infections like meningitis, pneumonia, and Haemophilus influenzae.

However, you would not be able to join if you have certain health issues that could make the study unsafe for you or make it difficult to get clear results. This includes current serious infections or cancer. Also, you cannot have had a history of recurrent serious infections caused by certain bacteria, or have had a stem cell transplant in the past.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of PNH?
Have you previously completed a Phase 2 or 3 study with iptacopan and were still taking it?
Does your doctor think you are still benefiting from iptacopan and have been stable on it for 3 months?
Have you had the required vaccinations (meningitis, pneumonia, Haemophilus influenzae)?
Have you NOT had a stem cell transplant?

Answer every question to see your result.

What does participation involve?

If you join this study, you will continue to receive iptacopan, the same medication you were taking in the previous study. You'll have regular visits to the clinic so the medical team can monitor your health, how you're feeling, and how the medication is working. These visits might include blood tests, health checks, and discussions about any side effects you might be experiencing.

This is an ongoing study, meaning there isn't a fixed end date for all participants. The total duration of your participation will depend on various factors, but the aim is to allow continued access to iptacopan for as long as it is beneficial and safe for you. The medical team will guide you through all the necessary assessments and follow-up appointments.

Potential risks and benefits

Participating in this study means you may continue to receive a treatment that has previously benefited you, potentially helping to manage your PNH symptoms for a longer period. However, like all medications, iptacopan can have side effects, and your health will be closely monitored for any potential risks. While the aim is to provide ongoing access to treatment, there's no guarantee that iptacopan will continue to be effective or won't cause new side effects over time. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (60)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

City Of Hope National Med Center
Verified postcode
Duarte, United States
City Of Hope
Verified postcode
Duarte, United States
USC Norris Cancer Center
Verified postcode
Los Angeles, United States
Univ Cali Irvine ALS Neuromuscular
Verified postcode
Orange, United States
Univ of California Irvine (Chao Family Comprehensive Cancer Center)
Verified postcode
Orange, United States
Lakes Research
Verified postcode
Miami Lakes, United States
Augusta University Georgia
Verified postcode
Augusta, United States
Augusta University
Verified postcode
Augusta, United States
Montefiore Medical Center
Verified postcode
The Bronx, United States
Cleveland Clinic Foundation
Verified postcode
Cleveland, United States
Prisma Health Upstate
Verified postcode
Greenville, United States
Novartis Investigative Site
Verified postcode
Santo André, Brazil

Common questions

What is PNH?

PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare blood disorder where red blood cells are destroyed too early, leading to symptoms like tiredness and anaemia.

What is iptacopan?

Iptacopan is a medicine being researched as a treatment for PNH to help manage the symptoms.

Why is this study only for people already taking iptacopan?

This study is a continuation to monitor the long-term safety and effects of iptacopan for people who have already found it helpful in earlier studies.

Will I have to pay to be in the study?

No, you will not have to pay for the iptacopan medication or any study-related tests and appointments if you participate.

How long will I be in the study?

This is an ongoing study for continued treatment, so there isn't a fixed end date. Your participation will continue as long as the treatment is beneficial and safe for you.