AuthorisedPhase I and Phase II (Integrated)- OtherInterventional

Phase I/II Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of VSA012 Injection in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Naïve or Have Not Received Complement Inhibitor Therapy Recently, as well as in Those with Persistent Anemia Despite Stable C5 Complement Inhibitor Therapy

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare condition where part of your body's natural defence system mistakenly attacks and destroys healthy red blood cells. This can lead to severe tiredness, anaemia, and a higher risk of dangerous blood clots. This early-stage study is testing an experimental injection called VSA012. Researchers want to see how safe the medicine is, how your body processes it, and whether it can effectively stop the breakdown of red blood cells. The study welcomes adults who have never had targeted PNH treatment, have not had it recently, or are already taking standard medicines but still experience persistent anaemia. By checking blood counts, energy levels, and heart health, the study team hopes to find out if VSA012 can help reduce the need for blood transfusions and improve daily quality of life.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Bisirna Therapeutics Pte. Ltd.

Enrolment target

Start

15 Oct 2026

Paroxysmal nocturnal hemoglobinuria (PNH)

What is this study about?

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, long-term blood disorder. In people with PNH, red blood cells lack normal protective shields. Because of this, an active part of the immune system called the complement system mistakenly destroys them. This early breakdown of blood cells causes severe anaemia, profound tiredness, dark urine, and an increased risk of blood clots.

While effective treatments exist to block this part of the immune system, they do not work perfectly for everyone. Some people still feel worn out, remain anaemic, or continue to need regular blood transfusions despite being on treatment. Other people have not yet had the chance to start modern therapies. This study is testing an experimental medicine called VSA012 to see if it can offer better, longer-lasting protection for red blood cells.

Because this is a Phase I/II trial, the main priority is safety. Doctors want to know what dose works best, what side effects might happen, and how the body breaks down the medicine. They will also look closely at early signs of benefit, such as rising red blood cell levels, fewer blood transfusions, and lower risk markers for blood clots.

Taking part in this research helps doctors understand whether VSA012 could become a reliable new treatment option for PNH in the future, giving patients more energy and better long-term health.

Key takeaways

Testing a new injection called VSA012 for adults with paroxysmal nocturnal haemoglobinuria (PNH).
Open to patients who are new to treatment, as well as those still experiencing anaemia on standard therapies.
Aims to check the safety of the medicine and see if it helps protect red blood cells and reduce fatigue.
Involves regular health monitoring, including blood tests and heart checks.
Participation is completely voluntary, and you can withdraw at any time.

Who may be eligible?

This trial is open to adults aged 18 and over who have been diagnosed with paroxysmal nocturnal haemoglobinuria (PNH). Both men and women can take part.

Doctors are looking for two main groups of patients. The first group includes people who have never taken complement inhibitor medicines before, or who have not received them recently. The second group includes people who are currently taking an approved complement medicine, but whose blood counts remain low, leaving them with ongoing anaemia.

You will need to undergo initial health checks to ensure it is safe for you to join. People with certain severe infections, other uncontrolled medical conditions, or allergies to similar medications may not be able to take part.

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.

I am aged 18 or older.
I have a confirmed diagnosis of paroxysmal nocturnal haemoglobinuria (PNH).
I have either never received complement inhibitor therapy (or not recently), OR I am currently on therapy but still have low blood counts (anaemia).
I am able and willing to attend regular hospital or clinic appointments for monitoring.
I am willing to receive injections and have routine health checks like blood tests and ECGs.

Answer every question to see your result.

What does participation involve?

If you take part, you will receive the study medicine, VSA012, as an injection. The study team will explain how often you will need the injections and how long your treatment will last. Throughout the study, you will have regular clinic visits so the team can monitor your health. These visits will include routine blood tests, heart checks (ECGs), physical examinations, and blood pressure measurements. You will also be asked to complete short questionnaires about your energy levels and daily fatigue. The team will closely follow your progress to see how your body handles the medicine and check for any side effects.

Potential risks and benefits

Potential benefits may include better control of your PNH, fewer blood transfusions, and an improvement in fatigue and anaemia, though these benefits cannot be guaranteed. Because VSA012 is an experimental medicine, there is a risk of unexpected side effects, reactions around the injection site, or a higher chance of certain infections because the medicine calms part of your immune system. Your health will be watched very closely throughout the trial. Taking part is completely voluntary, and you are free to leave the study at any time without your regular NHS care being affected.

Locations (4)

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

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Unverified
Spain
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Unverified
Germany
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Unverified
Italy
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Unverified
France

Common questions

What is PNH?

PNH stands for paroxysmal nocturnal haemoglobinuria. It is a rare blood disorder where the immune system attacks and destroys red blood cells, causing severe tiredness, anaemia, and other health issues.

How is the study medicine given?

VSA012 is given as an injection. The medical team will provide exact details on how often and where the injection is given.

Can I leave the study if I change my mind?

Yes. Taking part is entirely your choice. You can leave the study at any stage, and your usual medical care will continue as normal.

Will taking part cost me anything?

No, the study medication and all trial-related tests are provided free of charge. Travel expenses to study visits are usually reimbursed.

Will this replace my current PNH treatment?

Depending on your group in the study, you may start VSA012 as a new treatment or add/switch to it under close medical supervision. The trial doctor will explain your exact plan.