RecruitingRare disease

Study of pegcetacoplan safety and effectiveness in adolescents aged 12-17 with paroxysmal nocturnal hemoglobinuria

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Paroxysmal Nocturnal Hemoglobinuria, a rare blood disorder where the immune system attacks and destroys red blood cells. The study will evaluate a medication called pegcetacoplan, given as a solution that is injected under the skin. This medicine works by helping to protect red blood cells from being destroyed by the body's immune system.

The purpose of this research is to understand how the medication works in young patients aged 12-17 years and to determine if it is safe and effective for treating their condition. The study will look at how the medication affects various aspects of the blood, including hemoglobin levels (a protein that carries oxygen in red blood cells) and other markers that show how well the treatment is working.

Participants will receive treatment with pegcetacoplan for 16 weeks, during which they will need regular check-ups to monitor their health and how well the medication is working. The medication will be given through injections under the skin. Throughout the study, doctors will closely monitor the participants' blood test results and any side effects that may occur. The total study duration may extend up to 52 weeks to gather long-term safety and effectiveness information.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment phase
The treatment begins with pegcetacoplan (ASPAVELI), given as a solution under the skin (subcutaneous use)
The medication will be administered at a dose of 1,080 mg
You or your caregiver will need to learn how to give the medication through subcutaneous injection
If not already vaccinated, you will receive vaccines against specific bacteria (Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae) within 14 days of starting treatment
Step 2
First 16 weeks of treatment
Regular blood tests will monitor your response to treatment
The tests will measure:
- Hemoglobin levels (a protein that carries oxygen in your blood)
- LDH levels (an indicator of cell damage)
- Red blood cell counts
Your overall health and any side effects will be monitored
Step 3
Extended treatment phase
Treatment continues for up to 52 weeks
Regular monitoring of your blood tests continues
Additional assessments include:
- Complement system activity (part of your immune system)
- Quality of life measurements
- Number of blood transfusions needed, if any
Your overall health and any side effects continue to be monitored
Step 4
Final evaluation
Assessment of your response to treatment over the full 52-week period
Final blood tests and health evaluations
Review of any side effects or complications that occurred during treatment

Who can join the trial?

14 criteria

Age must be between 12 and 17 years old at the start of the study
Must weigh at least 20 kilograms
Must have a body mass index (BMI) below the 95th percentile for their age
Must have confirmed diagnosis of PNH through special blood testing showing specific cell populations above 10%
Must have either:
- Never received complement inhibitor treatment with low blood hemoglobin and high LDH levels, or
- Currently be on stable complement inhibitor treatment with low blood hemoglobin
- Blood test requirements:
  - Platelet count above 75,000 per cubic millimeter
  - Neutrophil count (type of white blood cell) above 1,000 per cubic millimeter
  - Must be willing and able to self-administer the medication or have a caregiver who can do it
  - Must be vaccinated against specific bacteria (Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae) or agree to receive vaccines within 14 days of starting treatment
  - For females who can become pregnant: must have negative pregnancy tests and use approved birth control methods during the study and for 90 days after
  - For sexually mature males: must use approved birth control methods and not donate sperm during the study and for 90 days after
  - Must maintain stable doses of any current medications (such as blood thinners, steroids, or supplements) for specified periods before starting the study
  - Must be willing to provide written informed consent (and assent if appropriate) to participate in the study

Who cannot join the trial?

15 criteria

Current or recent (within 3 months) pregnancy or breastfeeding
History of severe allergic reactions to any medications
Active, uncontrolled bacterial infections
Severe liver problems (as shown by blood tests)
Major surgery in the past 3 months
Participation in other clinical trials within the last 30 days
Active cancer or cancer treatment
Severe heart conditions or uncontrolled high blood pressure
History of blood clotting disorders
Inability to follow study procedures or attend scheduled visits
Use of certain medications that might interact with the study drug
Severe kidney disease (as determined by blood tests)
Mental conditions that could affect ability to provide informed consent
Drug or alcohol abuse within the past year
Any condition that, in the opinion of the study doctor, would make participation unsafe

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Hospital Universitario 12 De Octubre
Verified
Madrid, Spain
Hospital Vall d’Hebron
Verified
Barcelona, Spain
Universitair Medisch Centrum Utrecht
Responsive
Utrecht, The Netherlands

Trial locations

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Investigated drugs

Pegcetacoplan is a medication designed to treat paroxysmal nocturnal hemoglobinuria (PNH). It works by targeting and regulating part of the body's complement system, which is a component of the immune system. This medication helps protect red blood cells from destruction in patients with PNH, potentially improving anemia and reducing the need for blood transfusions. It is administered under the skin (subcutaneously) and is being studied specifically in adolescent patients to understand how it works in younger populations.

Pegcetacoplan

What is already known about the treatment

Pegcetacoplan - A novel therapeutic agent administered subcutaneously for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. This medication works by targeting and inhibiting the complement protein C3, which helps prevent the destruction of red blood cells characteristic of PNH. As a complement inhibitor, pegcetacoplan represents a significant advancement in PNH treatment, showing promising results in clinical trials for maintaining healthy hemoglobin levels and reducing the need for blood transfusions. The medication is currently being studied in pediatric populations, particularly adolescents, to evaluate its safety profile, pharmacokinetics, and effectiveness in managing PNH symptoms.

Investigated diseases

Paroxysmal Nocturnal Hemoglobinuria - A rare blood disorder where the immune system attacks and destroys red blood cells. The condition occurs due to a genetic mutation that affects the protective coating of blood cells. The destruction of red blood cells happens continuously but often becomes worse during sleep, leading to the presence of dark-colored urine in the morning. The condition causes anemia and can result in fatigue, weakness, and shortness of breath. The disease typically develops in young adulthood but can occur at any age.