Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A Phase 2 Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in PNH Patients with a Sub-optimal Response to the C5 inhibitor, Ravulizumab

This research study is looking at a new investigational medication called OMS906 for adults who have a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). PNH can cause red blood cells to break down, leading to symptoms like tiredness and anaemia. This study is specifically for people with PNH who are already being treated with a medicine called ravulizumab, but whose condition isn't fully controlled by it. Researchers want to understand if OMS906 is safe, how the body handles it, and if it could be a helpful additional treatment option. This is a very early stage of testing in humans, aiming to gather initial information about this new potential medicine.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Omeros Ireland Limited, Omeros Corp.

Enrolment target

Start

14 Nov 2022

Paroxysmal nocturnal hemoglobinuria

What is this study about?

This clinical trial is about a new medicine called OMS906. It's being developed for people living with a rare blood disorder called paroxysmal nocturnal hemoglobinuria, or PNH. PNH is a condition where some of your red blood cells are more fragile and break down too easily. This can lead to symptoms like feeling very tired, having low energy, and needing blood transfusions.

Currently, many people with PNH are treated with medicines that work by blocking a part of their immune system called C5. One common medicine is ravulizumab. While ravulizumab works well for many patients, for some, it doesn't completely control their PNH symptoms or improve their blood counts as much as doctors and patients would hope. So, this study is looking at OMS906 as a potential new option for these individuals.

The main goals of this study are to see if OMS906 is safe to use and how well people tolerate it. Researchers will also be looking at how the body processes the medication and if it starts to show any positive effects on PNH symptoms for those whose current treatment isn't fully effective. This is an important step in potentially offering new ways to manage PNH.

Key takeaways

This study is testing a new medicine for PNH.
It's for adults whose current PNH treatment isn't fully effective.
The main goals are to check safety and if it helps PNH.
Participation involves regular clinic visits and close monitoring.
This is an early stage of research for a potential new treatment.

Who may be eligible?

To join this study, you must be an adult aged 18 or over. This study is for both men and women.

Crucially, you would need to have been diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Another important requirement is that you are currently receiving treatment for PNH with a medicine called ravulizumab, but your PNH symptoms or blood test results show that this treatment isn't working as well as it could be for you.

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 Paroxysmal Nocturnal Hemoglobinuria (PNH)?
Are you currently being treated with the medicine ravulizumab for your PNH?
Do you feel that your PNH is not fully controlled by your current ravulizumab treatment?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the clinic for medical check-ups and assessments. You would receive the investigational medicine, OMS906, according to the study plan. These visits will include blood tests to check your PNH, see how the medicine is working, and monitor your general health. You’ll also have physical exams and discussions about any side effects you might be experiencing. The total duration of your participation in the study, including follow-up after your last dose, will be explained in detail by the study team.

Potential risks and benefits

Potential benefits could include an improvement in your PNH symptoms or blood test results if OMS906 proves effective, especially if your current treatment isn't fully working. However, as with any new medicine, there are potential risks, including side effects that are not yet fully known. The study team will explain all known and potential risks in detail. It's very important to remember that you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

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

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Unverified
Greece
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Unverified
Germany

Common questions

What is PNH?

PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare condition where red blood cells break down too quickly, leading to symptoms like tiredness and anaemia.

What does 'sub-optimal response' mean?

It means your current PNH treatment (ravulizumab) isn't fully controlling your condition or improving your symptoms as much as doctors would like.

Is OMS906 a new medicine?

Yes, OMS906 is an investigational medicine, meaning it's still being tested in clinical trials and is not yet approved for general use.

Will I still take my usual PNH medicine?

The study will provide detailed instructions on how OMS906 interacts with your current medication. The researchers will explain this clearly.

What if I get side effects?

The study team will monitor you closely for any side effects and provide care and support. You should report any changes in how you feel immediately.