Not recruitingRare disease

Study on the Effectiveness and Safety of Iptacopan for Adults with Atypical Hemolytic Uremic Syndrome Not Previously Treated with Complement Inhibitors

Verified siteInvestigationalNo placebo

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What is this trial about?

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

This clinical trial is focused on studying a condition called atypical hemolytic uremic syndrome (aHUS). aHUS is a rare disease that affects the blood and kidneys, leading to problems like low platelet counts, destruction of red blood cells, and kidney damage. The treatment being tested in this study is a medication called iptacopan, which is taken orally in the form of hard gelatin capsules. Iptacopan is also known by its code name, LNP023.

The purpose of the study is to evaluate the effectiveness and safety of iptacopan in adult patients with aHUS who have not previously received a type of treatment known as complement inhibitor therapy. Participants in the study will take iptacopan twice daily for a period of 26 weeks. During this time, researchers will monitor the participants to see if they achieve a complete response in their condition, which means improvement in blood and kidney function without the need for other treatments like plasma exchange or specific antibodies.

The study will also look at how quickly participants respond to the treatment, changes in blood parameters, kidney function, and overall health and well-being. Safety evaluations will be conducted to monitor any side effects or adverse events. The study aims to provide valuable information on the potential benefits of iptacopan for individuals with aHUS.

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
Joining the study
Upon joining the study, ensure that all necessary vaccinations are completed. This includes vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae, if not previously received. Vaccinations should be administered at least 2 weeks before starting the study medication. If immediate start is necessary, antibiotics may be prescribed for at least 2 weeks post-vaccination.
Step 2
Medication administration
The study involves taking iptacopan, which is provided in the form of hard gelatin capsules. The medication is taken orally, twice daily. The duration of the treatment is 26 weeks.
Step 3
Monitoring and assessments
Throughout the 26-week period, regular monitoring will occur to assess the response to the treatment. This includes checking blood parameters such as platelet count, lactate dehydrogenase (LDH), and hemoglobin levels. Kidney function will also be evaluated by measuring serum creatinine levels.
The goal is to achieve a complete response, which involves normalization of blood parameters and improvement in kidney function. This response must be maintained for at least two measurements taken four weeks apart.
Step 4
Evaluation of outcomes
At the end of the 26 weeks, various outcomes will be evaluated. These include changes in blood parameters, kidney function, and patient-reported outcomes related to fatigue and overall health status.
Safety evaluations will also be conducted, including monitoring for any adverse events or serious adverse events.

Who can join the trial?

8 criteria

Participants must be **18 years or older** and have signs of **active thrombotic microangiopathy (TMA)**. TMA is a condition that affects blood cells and the kidneys.
Participants must have a **low platelet count** (less than 150,000 per microliter) during the screening period. Platelets are cells that help with blood clotting.
Participants must have **high levels of LDH** (lactate dehydrogenase) in the blood, at least 1.5 times the normal upper limit, during the screening period. LDH is an enzyme that can indicate tissue damage.
Participants must have **low hemoglobin levels** during the screening period. Hemoglobin is a protein in red blood cells that carries oxygen.
Participants must have **high serum creatinine levels** during the screening period, indicating worsening kidney function. Serum creatinine is a waste product in the blood that comes from muscle activity.
Participants must be vaccinated against **Neisseria meningitidis** and **Streptococcus pneumoniae** infections before starting the study treatment. If not previously vaccinated, the vaccine should be given at least 2 weeks before starting the study drug. If the study treatment starts earlier, antibiotics must be given at the start of the study treatment and for at least 2 weeks after vaccination.
If not previously vaccinated, participants should receive a vaccine against **Haemophilus influenzae** infection, if available, at least 2 weeks before starting the study drug.
For participants with a **kidney transplant**, they must have a known history of atypical hemolytic uremic syndrome (aHUS) before the current transplant. If no history is available, they may still be eligible if other causes of TMA are ruled out, especially transplant rejection. If changes to immunosuppressive medications are needed after transplantation, TMA must persist for at least 4 days after the change.

Who cannot join the trial?

10 criteria

Patients who have a history of severe allergic reactions to the study medication.
Patients who are currently participating in another clinical trial.
Patients with a history of drug or alcohol abuse within the past year.
Patients who are pregnant or breastfeeding.
Patients with any other medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who have received certain medications that might interfere with the study treatment.
Patients with a history of certain types of cancer.
Patients with severe liver or kidney disease.
Patients who have had a recent major surgery.
Patients with uncontrolled high blood pressure.

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Verified sites

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

Institute For Clinical And Experimental Medicine
Verified
Prague, Czechia
Laiko General Hospital Of Athens
Verified
Athens, Greece
Medical University Of Vienna
Verified
Vienna, Austria

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Investigated drugs

Iptacopan is an oral medication taken twice daily. It is being studied for its effectiveness and safety in adult patients with a condition called atypical Hemolytic Uremic Syndrome (aHUS) who have not previously received treatment with complement inhibitor therapy. The main goal of the trial is to see how well iptacopan can help these patients achieve a complete response in reducing the symptoms of thrombotic microangiopathy (TMA) over a period of 26 weeks.

Iptacopan

What is already known about the treatment

Iptacopan – Iptacopan is administered orally in the form of tablets, taken twice daily. It is currently being studied in clinical trials for its effectiveness and safety in treating atypical hemolytic uremic syndrome (aHUS) in patients who have not previously received complement inhibitor therapy. The main therapeutic indication for iptacopan is to manage aHUS, a condition characterized by abnormal blood clotting and kidney damage. At the molecular level, iptacopan works by inhibiting a component of the complement system, which is part of the immune system that can contribute to the disease process in aHUS. It is classified pharmacologically as a complement inhibitor.

Investigated diseases

Atypical Hemolytic Uremic Syndrome – Atypical hemolytic uremic syndrome is a rare condition characterized by the destruction of red blood cells, leading to kidney failure and low platelet count. It is caused by the abnormal activation of the immune system, which results in the formation of blood clots in small blood vessels. These clots can damage organs, particularly the kidneys, leading to symptoms such as fatigue, high blood pressure, and reduced urine output. The disease can progress with episodes of worsening symptoms, often triggered by infections or other stressors. Over time, the repeated damage to the kidneys can lead to chronic kidney disease. The condition requires careful management to prevent complications and preserve kidney function.