RecruitingPhase IInterventional

Safety and tolerability of APL-3007 administered as a single dose in addition to background therapy with a C5 inhibitor in adults with paroxysmal nocturnal hemoglobinuria

This research study is looking at a new medicine called APL-3007. It's for adults who have a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). Even though they are already being treated with a standard PNH medicine (a C5 inhibitor), their condition isn't fully controlled. The study aims to find out if APL-3007 is safe to use and how well people can tolerate it when given as a single dose alongside their existing treatment. This is a very early stage (Phase 1) study, meaning we are primarily focused on understanding any side effects and how the body reacts to the new medicine, rather than whether it cures the condition. Your participation could help researchers learn more about new ways to manage PNH.

At a glance

Status

Recruiting

Phase

Phase I

Sponsor

Apellis Pharmaceuticals (United States)

Enrolment target

Start

20 Nov 2025

Estimated completion

30 Mar 2027

Paroxysmal Nocturnal Hemoglobinuria (PNH)

What is this study about?

Imagine your body has tiny, important cells in your blood, called red blood cells. In a condition called Paroxysmal Nocturnal Hemoglobinuria, or PNH, these red blood cells can break down too quickly, causing problems like tiredness and anaemia. Many people with PNH are already taking a medicine called a C5 inhibitor, which helps to stop some of this breakdown.

This study is looking at a new medicine, APL-3007, to see if it can help adults with PNH whose condition isn't fully controlled by their current C5 inhibitor treatment. It's an early-stage study, called Phase 1, which means the main goal is to carefully check if the new medicine is safe and if people taking it experience any side effects. Researchers will also look at how the body handles the medicine.

Think of this as an important first step. If APL-3007 proves to be safe and well-tolerated, it could potentially be developed further to become another option for people living with PNH. Your involvement could help scientists gather crucial information that might lead to new and better treatments in the future.

Key takeaways

Tests a new medicine (APL-3007) for PNH.
For adults already on a C5 inhibitor for PNH but still having symptoms.
Focuses on safety and how well the medicine is tolerated.
Participation includes one dose of APL-3007 alongside existing treatment.
Your involvement helps PNH research for the future.

Who may be eligible?

To join this study, you need to be an adult, aged between 18 and 70 years old, and have been diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). You should have been on your current PNH treatment (a C5 inhibitor called ravulizumab-cwvz) for at least six months, and despite this, your blood tests still show signs that your PNH isn't fully controlled, such as low haemoglobin levels (below 10.5 g/dl) and signs of red blood cell breakdown.

There are also specific requirements for women. If you are female, you must either be unable to get pregnant (for example, if you've gone through menopause and haven't had a period for over a year, or have had certain surgeries) or, if you can get pregnant, you must have a negative pregnancy test before starting. If you can get pregnant, you would need to agree to use effective birth control methods throughout the study and not breastfeed.

Finally, you would also need to have proof of having received vaccinations against certain infections like pneumonia and meningitis. The study team will review all your medical information carefully to see if you meet all the requirements.

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 to 70 years old?
Do you have a PNH diagnosis?
Have you been on ravulizumab-cwvz for at least 6 months, but still have low haemoglobin (below 10.5 g/dl) and other signs of PNH activity?
If you are a woman who can get pregnant, are you willing to use effective birth control and not breastfeed during the study?
Do you have proof of vaccination against pneumonia and meningitis?

Answer every question to see your result.

What does participation involve?

Taking part in this study means you would receive a single dose of the study medicine, APL-3007, in addition to your usual PNH treatment. The study team would closely monitor you before, during, and after you receive this dose. This would involve regular visits to the clinic for medical check-ups, blood tests, and to answer questions about how you are feeling. The total duration of your active participation in the study, including follow-up checks after receiving the medicine, would be explained in detail by the study team. They will ensure you understand what each visit involves and how long your commitment would be.

Potential risks and benefits

While we hope this study will provide valuable information about a potential new medicine for PNH, it's important to understand there might not be direct medical benefits to you personally, as the main goal is to assess safety. However, your participation contributes significantly to medical research and could help others with PNH in the future. As with any new medicine, there are potential risks, including side effects from APL-3007 itself or from the procedures involved (like blood tests). The study team will explain all known and potential risks in detail. Remember, joining a study is always your choice, and you have the right to withdraw at any time without affecting your standard medical care.

Locations (1)

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

Leeds Teaching Hospitals NHS Trust
City only
Leeds, England

Common questions

What is PNH?

PNH is a rare blood condition where your red blood cells break down too quickly, which can cause symptoms like tiredness.

What is the new medicine, APL-3007, for?

APL-3007 is a new medicine being tested to see if it's safe to use for adults with PNH whose condition isn't fully controlled by their usual treatment.

Will I stop my current PNH treatment?

No, you will continue taking your current PNH medicine (a C5 inhibitor) as usual. APL-3007 would be given in addition to it.

Why is this study only for 18 to 70-year-olds?

Clinical trials often set age ranges to focus on a specific group where the medicine's effects are most relevant and safe to study initially.

What does 'Phase 1' mean?

Phase 1 means this is an early study primarily focused on checking the safety of the new medicine and how the body handles it, rather than its effectiveness.

How to find out more

- Apellis Pharmaceuticals

clinicaltrials@apellis.com +44 01908 251 480 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.