A Phase 3, Open Labelled study in children and adolescents with PNH
Research type
Research Study
Full title
A PHASE 3, OPEN-LABEL STUDY OF ALXN1210 IN CHILDREN AND ADOLESCENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
IRAS ID
235914
Contact name
Austin Kulasekararaj
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2017-002820-26
Duration of Study in the UK
3 years, 7 months, 3 days
Research summary
Research Summary
This study is a phase 3 open-labelled study of ALXN1210 (study drug) for children and adolescents with Paroxysmal Nocturnal Haemoglobinuria (PNH). PNH is a rare blood condition where blood cells are prone to be attacked by part of the body’s immune system. It is an extremely rare condition and only 1-2 people per million worldwide are diagnosed each year. PNH can develop at any age with most patients being diagnosed in their 30s or 40s; children and older patients can also develop the condition.
Currently, eculizumab (a precursor to ALXN1210 marketed under the brand name Soliris®) is the standard of care treatment for patients with PNH who need therapy. The expected benefit of ALXN1210 over eculizumab is the dosing frequency; while eculizumab is delivered by intravenous (IV) infusion every 2 weeks, ALXN1210 is being tested for dosing to occur every 8 weeks. This has the potential to significantly improve the quality of life for patients on treatment.
The purpose of this study is to assess pharmacokinetics (what the body does with the drug), pharmacodynamics (how the body reacts to the drug), safety, and efficacy in children with this disease.The study will be in 3 periods, Screening (4 weeks), Treatment (26 weeks) and Extension (up to 2 years). Eligible participants are expected to be in the study for approximately 2.5 years. It is expected that approximately 12 participants will be recruited globally to take part in the study.
The study drug will be administered intravenously (into a vein) and the participant’s condition will be carefully monitored throughout the study. Other study procedures include physical examinations, blood and urine samples, vital signs and ECG.
Summary of Results
CLINICAL STUDY RESULTS A Study to Learn How Ravulizumab Works and if it is Effective and Safe in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria
What are clinical studies?
Clinical studies, also called clinical trials, aim to answer specific questions about new or existing treatments, procedures, or vaccines and involve participants with health conditions or healthy volunteers. For research on a new treatment, clinical studies happen in several stages, from Phase 1 to Phase 4, depending on the specific question to be investigated in the study. This was a Phase 3 study. Many Phase 3 studies look at the overall risks and benefits of a new treatment compared to a placebo (a product similar in appearance to the treatment being tested but contains no real medicine) or a control group (a group of participants who are not given the treatment in the study but serve as a reference for future comparison).In this study, a drug called ravulizumab was tested. The researchers wanted to know how ravulizumab works in children and adolescents with paroxysmal nocturnal hemoglobinuria (also known as PNH), by measuring its level and its activity in the blood over time. The researchers also wanted to know how safe ravulizumab was by looking at any side effects occurring in the participants.
What is PNH?
PNH is a very rare, life-threatening but treatable blood disorder in which some or all of the red blood cells (cells that carry oxygen around the body) are defective and lack important protective proteins that prevent the breakdown of the red blood cells.
Without these protective proteins, the body’s natural defense system, also called the immune system, destroys the red blood cells. This process is known as hemolysis. For people living with PNH, hemolysis is always happening and can result in serious health problems. During hemolysis, hemoglobin (the protein contained in red blood cells that carries oxygen around the body) is destroyed and lactate dehydrogenase (also known as LDH) is released into the blood; the levels of LDH in the blood indicate how much hemolysis is taking place. High levels of LDH are a typical sign of PNH.What are the symptoms of PNH?
The most common symptoms of PNH include feeling tired, difficulty swallowing, trouble concentrating or thinking, shortness of breath, stomach pain, dark-colored urine, and erectile dysfunction (difficulty in getting and keeping an erection) in men.The lack of healthy red blood cells, known as anemia or being anemic, is a serious complication of PNH, which often results in patients needing blood transfusions. Life-threatening complications from PNH can include blood clots, kidney failure, and other organ damage.
What treatments are available for PNH?
Ravulizumab, eculizumab and pegcetacoplan are approved treatments for PNH. These treatments work by inhibiting (blocking the function of) the complement system, an important part of the immune system (the body’s natural defences).Ravulizumab is a monoclonal antibody (protein used by the immune system) produced in cells, and provided as an infusion for use in humans. When administered to people, ravulizumab inhibits the complement system. The complement system is composed of more than 40 proteins, including a protein called C5. Ravulizumab works by sticking to the C5 protein. This action helps stop the red blood cells from breaking down and being destroyed.
Results of this study contributed to the approval of the use of ravulizumab in children. Supportive therapies used before the approval of ravulizumab for children included blood transfusions and blood thinners.
What were the treatments researched in this study?
In this study, participants received a drug called ravulizumab. Ravulizumab was given intravenously (IV; administered through a vein into the bloodstream).During the Treatment Period (26 weeks), participants were given “open-label” ravulizumab, which means that the participants, participants’ caregivers, and the study doctors knew which treatment was received. After the Treatment Period, participants could continue the treatment with ravulizumab in the Extension Treatment Period (up to 4 years, or until ravulizumab was registered or approved).
Participants were given a personalized dose of ravulizumab based on their body weight. Participants started with a loading dose (initial dose of the study drug, usually followed by a lower dose) of ravulizumab on Day 1. This dose was followed by a maintenance dose on Day 15. After Day 15, the maintenance dose was given once every 4 weeks to participants weighing less than 20 kilograms (kg) and once every 8 weeks to participants weighing 20 kg or more.
Participants were assigned to two groups:
• Group 1: participants who never took eculizumab (or any other inhibitor of the complement system).
• Group 2: participants who previously received eculizumab.Participants who were treated with eculizumab (a different inhibitor of the complement system) were asked to stop taking eculizumab 2 weeks before receiving the loading dose of ravulizumab.
Who could take part in this study?
To be able to take part in the study, participants had to meet the following requirements:
• Male or female children and adolescents (less than 18 years old)
• Confirmed diagnosis of PNH
• At least one sign or symptom of PNH in participants not previously treated with eculizumabIndividuals were unable to take part if study doctors determined they had a low count of platelets (components that help the blood to clot) or neutrophils (a type of white blood cell) in the blood. Also, those who had undergone a transplant of bone marrow or who had a history of recurrent infections could not take part in the study.
Complement inhibitors like ravulizumab increase the risk of potentially life-threatening meningococcal infection, which is caused by a bacterium called Neisseria meningitidis. To help prevent it, all participants had to be adequately vaccinated with meningococcal vaccine before receiving ravulizumab.
A parent or guardian provided consent for participants under 18 years of age to participate in this study. Where applicable, children and young adults under 18 years of age could also provide their assent to participate in this study.
How many participants took part in this study?
5 boys + 8 girls = 13 participants
Participants were between 9 and 17 years of age at the start of the study. The study started in February 2018 and ended in August 2022.WHERE WAS THIS STUDY DONE? The study took place in 6 countries across 9 study centres in France, Netherlands, Norway, Russia, United Kingdom and the United States.
What were the results of this study?
The researchers wanted to see how the level of ravulizumab in the blood varied before and after the infusions, and if it was sufficient for its beneficial effect over time. To answer these questions, researchers assessed the following parameters:
1. the maximal level of ravulizumab (also known as Cmax), measured immediately after its infusion; 2. the level of ravulizumab measured between infusions (also known as Ctrough).
From the results obtained, researchers could calculate if the level of ravulizumab was sufficient to maintain its therapeutic effect throughout the Treatment Period. Researchers also checked that the level of ravulizumab was not too high to ensure there was no unexpected accumulation of ravulizumab.Researchers also verified the therapeutic activity of ravulizumab by testing the inhibition of the complement system and the reduction of hemolysis (disruption of red blood cells).
How did the levels of ravulizumab change over time? Did ravulizumab accumulate in the blood of participants during the Treatment Period?
Overall, the results showed that ravulizumab reached its maximal level in the blood immediately after its infusion and then decreased until the next infusion. This was observed throughout the Treatment Period.The maximal level achieved was considered safe and not associated with any accumulation (build up), while the level in between infusions was considered sufficient for the therapeutic effect of ravulizumab.
Did ravulizumab inhibit the complement system? Was the inhibition maintained throughout the Treatment Period?
Overall, the results showed that ravulizumab completely inhibited (caused 100% inhibition of) the complement system in all participants.
The inhibition happened immediately after the first infusion and was maintained throughout the Treatment Period in both Group 1 and Group 2.Did ravulizumab reduce hemolysis? Was the reduction in hemolysis maintained throughout the Treatment Period?
Overall, the results showed that hemolysis was inhibited by more than 80% on average in all participants. Hemolysis was inhibited immediately after the first infusion and was maintained throughout the Treatment Period in both Group 1 and Group 2.What were the other results of this study?
The researchers wanted to answer other important questions during the studyWhat was the change in LDH levels from the start of the study (baseline) to the end of the Treatment Period (Week 26)?
Results showed that ravulizumab decreased the levels of LDH by half in Group 1. In Group 2, ravulizumab maintained the levels of LDH stable between baseline and Week 26.How many participants did not need a blood transfusion during the study?
Overall, 11 out of 13 participants (84.6%) did not need any blood transfusion and remained transfusion-free throughout the Treatment Period. Of these, 3 participants were in Group 1 (60%) and 8 participants were in Group 2 (100%).Did ravulizumab have an effect on the participants’ hemolysis?
During the study, the researchers took blood samples from each participant and performed specific tests. These tests helped the researchers to understand more about red blood cells, blood clotting, and specific blood proteins in patients with PNH. In particular, researchers measured:
1. the number of participants who had stable levels of hemoglobin in the blood throughout the Treatment Period; 2. the change in levels of hemoglobin during the Treatment Period; 3. the number of participants having one or more breakthrough hemolysis (a serious episode of hemolysis causing a worsening of PNH symptoms) during the Treatment Period.Overall, the results showed that the treatment with ravulizumab reduced hemolysis in the blood:
1. 9 out of 13 participants had stable levels of hemoglobin in the blood throughout the Treatment Period; of these, 3 participants were in Group 1 and 6 participants were in Group 2; 2. at the end of the Treatment Period, the levels of hemoglobin were increased in Group 1 while they were generally stable in Group 2; 3. none of the participants experienced breakthrough hemolysis during the Treatment Period.Did ravulizumab improve the participants’ overall quality of life?
Researchers wanted to know if ravulizumab changed the quality of life of participants (how the participants felt about their own general wellbeing while taking ravulizumab). The quality of life was measured only in participants of 5 years of age or older using a specific patient-reported questionnaire.Overall, during the Treatment Period the quality of life was improved in participants in Group 1 and was unchanged in participants in Group 2. Importantly, at the beginning of the study, participants in Group 2 who were previously treated with eculizumab had a better quality of life than participants in Group 1.
What were the safety findings in this study?
A side effect is any symptom a participant has during the study which may or may not be related to the study treatment. Related side effects are unwanted medical events that happen during the study, and are considered to be related to study treatment. Side effects are classified as either “mild”, “moderate”, or “severe” in intensity.A serious side effect is thought to be an important medical event (e.g., requires a person to be admitted to the hospital, is life-threatening, causes disability, or causes death).
Side effects can vary from person to person. Researchers keep a record of all the side effects participants have when new treatments are studied. This helps determine which side effects occur as a result of the study treatment and which occur by chance or because of the participant’s underlying disease.
What serious side effects did participants have in this study?
Overall, 4 out of 13 participants (30.8%) had serious side effects during the entire study (Treatment Period + Extension Treatment Period), the majority of which were thought by study doctors to be not related to ravulizumab. Of those, 2 participants were in Group 1 (40%) and 2 participants were in Group 2 (25%). A total of 1 out of 13 participants (7.7%) had a side effect that was thought by the study doctor to be related to ravulizumab. The participant was in Group 2 (12.5%) and experienced breakthrough hemolysis.None of the participants died during the study.
What side effects did participants have in this study?
Overall, 13 out of 13 participants (100%) had side effects during the entire study (Treatment Period + Extension Treatment Period). A total of 6 out of 13 participants (46.2%) had a side effect that was thought by the study doctor to be related to ravulizumab. Of those, 1 participant was in Group 1 (20%) and 5 participants were in Group 2 (62.5%).The most common related side effect was anemia (lack of healthy red blood cells), which happened in 2 participants in Group 2 (25%).
Were there any other important safety findings in this study?
None of the participants stopped taking ravulizumab because of side effects.OUTCOME OF THE STUDY
How has this study helped patients and researchers?
The information collected in this study showed that the level of ravulizumab in the blood of children and adolescents with PNH was safe and sufficient to guarantee its therapeutic effect throughout the Treatment Period. Ravulizumab inhibited the complement system, reduced hemolysis and the need of blood transfusions and improved participants’ quality of life. The majority of side effects were thought by study doctors to be not related to ravulizumab.Before a treatment can be approved for patients to use, researchers look at the results of many studies to decide which treatments work best and are safe. If you have any questions about ravulizumab for the treatment of PNH please talk to your doctor. You should not change your treatment based on the results of this study without talking to a doctor first.
Useful clinical study websites
This document provides a summary of the main results of the study. It includes information about the side effects that happened to participants in the study and the results of the questions the researchers wanted to answer. This summary was reviewed for readability by a patient advocacy group.Complete study results are available to read at the following clinical study register(s):
https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C01%7Capprovals%40hra.nhs.uk%7C71a08727327d41f7c24208db3b3f726a%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638168915907639053%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=tVXaAkWaw37t6g0nrcMdzT4hgfaSCVeeAM7hAQCgt7w%3D&reserved=0
Use the study number NCT03406507 to search for more information on this website.
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Use the study number 2017-002820-26 to search for more information on this website.Further studies
PNH registries are ongoing. The “Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry” and “The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry” are programs that collect information on patients with PNH. In addition, several studies investigating ravulizumab for the treatment of PNH are either currently running or completed.
Please follow the links below to find out more information:
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3 Feb 2023. This summary includes known facts as of the time the document was finalized.REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
17/YH/0385
Date of REC Opinion
23 Nov 2017
REC opinion
Further Information Favourable Opinion