Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria
This study is looking into a new medication called crovalimab. We want to understand if it's safe, how the body handles it, and if it can help people who have a rare blood disorder called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a serious condition where red blood cells are destroyed too early. The study is divided into several parts. First, we'll give crovalimab to healthy volunteers to check its safety and how it behaves in their bodies. After that, we'll test it on people with PNH to see if it's safe, works well, and improves their condition. We are also comparing it to a placebo (a dummy treatment). The information gathered will help us understand if crovalimab could be a new treatment option for PNH.
At a glance
What is this study about?
This study is an important step in developing a new treatment for a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH. PNH is a serious illness where the body's immune system mistakenly attacks and destroys its own red blood cells. This can lead to tiredness, anaemia, and other health issues. The new medicine being tested is called crovalimab.
The main goals of this study are to find out if crovalimab is safe for people to use, how the body processes the medication, and if it's effective in treating PNH. Efficacy means whether it actually helps to improve the condition. The study is divided into different stages, starting with healthy volunteers and then moving on to people who have PNH.
By carefully studying crovalimab, we hope to gain a better understanding of its potential as a new treatment. This information will help doctors decide if crovalimab could offer a good treatment option for patients with PNH in the future.
Key takeaways
- This study is for a new PNH treatment called crovalimab.
- It checks if crovalimab is safe and effective.
- Both healthy volunteers and people with PNH can participate.
- Meningitis vaccinations are important for participation.
- You might receive the new medicine or a placebo.
- You can stop participating at any time.
Who may be eligible?
To join the first part of this study, you need to be a healthy man between 21 and 55 years old. You must be free from certain infections like Hepatitis B, Hepatitis C, and HIV. You also need to have had all your vaccinations for Hepatitis B and against certain types of meningitis. Participants in this part should not smoke, or have quit smoking at least two months before joining.
For the later parts of the study, we are looking for men and women with PNH, aged between 18 and 75 years. It's really important that you are up-to-date with your meningitis vaccinations according to local health guidelines. In some cases, if you're not fully vaccinated or if there's a short time between vaccination and starting the study medicine, you might need to take antibiotics to prevent meningitis.
If you are currently taking other medications for PNH, like immunosuppressants, steroids, or iron supplements, these treatments need to have been at a steady dose for at least four weeks before you can join. Some parts of the study are specifically for people with PNH who haven't been treated with certain types of PNH medications before, or who are looking to change their current treatment.
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 between 18 and 75 years old?
- Do you have a diagnosis of PNH?
- Are your meningitis vaccinations up-to-date, or are you willing to get them?
- If you're on other PNH medicines, has your dose been stable for at least 4 weeks?
- Are you willing to attend regular clinic visits?
What does participation involve?
If you decide to take part in this study, you'll be involved in several steps. Initially, healthy volunteers will receive a single dose of crovalimab, carefully monitored for safety. Later, for people with PNH, you'll receive multiple doses of either crovalimab or a placebo (a dummy treatment). This will involve regular visits to the clinic for medical checks, blood tests, and to receive your study medication.
Throughout the study, doctors and nurses will closely monitor your health, looking for any side effects and how your body reacts to the treatment. There will be various assessments to see if the medication is working and how it's affecting your PNH. The total duration of your participation will depend on which part of the study you are in, but it will involve ongoing follow-up to ensure your well-being.
Potential risks and benefits
Locations (14)
- Institut hematologie Centre Hayem CHU paris Saint-Louis Lariboisiere F Widal Hopital St LouisVerified postcodeParis, France
- Uniklinik RWTH AachenVerified postcodeAachen, Germany
- Universitätsklinikum EssenVerified postcodeEssen, Germany
- Universitätsklinikum UlmVerified postcodeUlm, Germany
- Semmelweis Egyetem, 1. Szamu Belgyogyaszati Klinika, DiabetologiaVerified postcodeBudapest, Hungary
- Kaposi Mor Teaching Hospital, Dept of Internal Medicine/HematologyVerified postcodeKaposvár, Hungary
- Policlinico Universitario Agostino GemelliVerified postcodeRome, Italy
- Tohoku University HospitalVerified postcodeMiyagi, Japan
- Osaka University HospitalVerified postcodeOsaka, Japan
- NTT Medical Center TokyoVerified postcodeTokyo, Japan
- Tokyo Medical University HospitalVerified postcodeTokyo, Japan
- University of Tsukuba HospitalVerified postcodeTsukuba, Japan
Common questions
What is PNH?
PNH, or Paroxysmal Nocturnal Hemoglobinuria, is a rare blood disorder where red blood cells are destroyed too early by the body's immune system.
What is crovalimab?
Crovalimab is a new medicine being tested in this study to see if it can help treat PNH.
What does 'placebo' mean?
A placebo is a 'dummy' treatment that looks like the real medicine but contains no active drug. It helps researchers compare the effects of the new medicine.
Will I know if I'm getting the real medicine or the placebo?
In some parts of the study, you might not know if you're receiving crovalimab or the placebo. This helps ensure fair results.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time, and this will not affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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