AN ADAPTIVE PHASE I/II STUDY TO ASSESS SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7112689 IN HEALTHY VOLUNTEERS AND PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood disorder where the immune system mistakenly destroys healthy red blood cells. This can cause severe tiredness, anaemia, and a need for frequent blood transfusions. This clinical trial is testing an experimental medicine called crovalimab. The study is split into stages: it first tests the drug in healthy volunteers to check safety, and then tests it in people diagnosed with PNH. In the early stage, some healthy volunteers may receive a dummy treatment (placebo) for comparison. Researchers want to see how safe the drug is, find the right dose, and check if it protects red blood cells. They will also track whether crovalimab improves energy levels and cuts down the need for blood transfusions. The ultimate goal is to find out if crovalimab could become a helpful, long-term treatment option.
En un coup d'œil
De quoi parle cette étude ?
Paroxysmal nocturnal haemoglobinuria, usually called PNH, is a rare condition that affects the blood. People with PNH have red blood cells that lack a protective coating. Because of this, an active part of the immune system called the complement system attacks and breaks them open. When red blood cells burst, it causes anaemia, overwhelming tiredness, dark-coloured urine, and a higher risk of dangerous blood clots.
This trial is testing a new treatment called crovalimab (also known as RO7112689). Crovalimab is designed to block the specific immune protein that attacks the red blood cells. This is an early-stage study (Phase I/II), meaning it is among the very first times the medicine is being tested in humans. The study starts by checking safety in healthy volunteers, and then moves on to test how well it works in people living with PNH.
Current standard treatments for PNH often require regular, time-consuming clinic visits for intravenous drips. Crovalimab has been designed so that it can be given as a simple injection under the skin (subcutaneous). Researchers want to find out how the body absorbs this injection, whether it effectively keeps the immune attack switched off, and if it helps people feel less tired in their daily lives.
By taking part, participants help doctors learn whether this medicine could offer a simpler, more effective way to control PNH symptoms and protect red blood cells from damage in the future.
Points clés
- Tests an experimental drug called crovalimab for paroxysmal nocturnal haemoglobinuria (PNH).
- Open to healthy volunteers (early stage) and adults diagnosed with PNH.
- Aims to protect red blood cells, ease fatigue, and reduce the need for blood transfusions.
- The medicine is designed to be given as an injection under the skin.
- Participation includes regular clinic visits, blood tests, and close safety checks.
Qui peut être éligible ?
This trial is open to adults aged 18 and older, including both men and women. Because this study has multiple parts, it is looking for two different groups of people at different stages: healthy adult volunteers and adults who have been diagnosed with PNH.
If you are joining as a healthy volunteer, you must have no major medical conditions. If you are joining as someone with PNH, doctors will check your medical history and blood tests to make sure your condition meets the study criteria, such as having signs that red blood cells are actively breaking down.
The study team will carry out a thorough health screening before you can join. People with certain serious infections, severe organ problems, or those taking specific medications that could clash with the study drug may not be able to take part.
Cet étude pourrait-elle vous convenir ?
Répondez à ces questions rapides pour voir si vous êtes éligible. Ceci est un guide uniquement — l'équipe de recherche prend la décision finale.
- I am aged 18 or older.
- I am either a healthy adult or have a confirmed diagnosis of PNH.
- I am willing to attend regular hospital or clinic appointments for tests.
- I am able to receive injections under the skin or through a drip.
- I am happy to complete questionnaires about my energy levels and health.
En quoi consiste la participation ?
Taking part involves several stages over a period of months. First, you will attend a screening visit where doctors check your overall health using blood tests, heart checks, and physical exams. If you join, you will receive crovalimab (or a placebo if you are a healthy volunteer in an early group) given as an injection under the skin or through a vein. You will need to attend regular clinic visits so doctors can take blood samples to measure haemoglobin levels, check for cell damage, and see how much medicine is in your bloodstream. You will also be asked to complete simple questionnaires about your energy levels and daily quality of life. After your treatment ends, there will be follow-up visits to check on your health and safety.
Risques et avantages potentiels
Emplacements (4)
- —Non vérifiéItalie
- —Non vérifiéAllemagne
- —Non vérifiéHongrie
- —Non vérifiéFrance
Questions fréquentes
What is PNH?
PNH is a rare blood disorder where the immune system attacks and destroys red blood cells, leading to anaemia, severe fatigue, and other health problems.
Could I receive a dummy drug (placebo)?
Placebos are only used in the early part of the study with healthy volunteers. People with PNH will receive the active study medicine.
Comment le médicament est-il administré ?
Crovalimab is designed to be given as an injection under the skin, though some initial doses may be given through a drip into a vein.
Can I leave the trial if I change my mind?
Yes. You can leave the study at any point without giving a reason, and your regular medical care will not be affected.
Does this trial replace my normal treatment?
This depends on the study part and your current care plan. The trial doctors will explain exactly how the study medicine fits alongside or replaces any existing treatments.
Comment en savoir plus
Parlez toujours à votre médecin généraliste ou spécialiste avant de décider de participer à une étude.
Intéressé à participer ?
Discussion
Discussion communautaire
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