RecruitingRare disease

Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

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 rare disease called Atypical Hemolytic Uremic Syndrome (aHUS). This condition affects the blood and kidneys, leading to problems like low platelet counts, destruction of red blood cells, and kidney failure. The study will explore the effects of a treatment called iptacopan, which is taken as hard gelatin capsules. Participants in the study will switch from their current treatment, an anti-C5 antibody therapy, to iptacopan.

The purpose of the study is to evaluate the safety and effectiveness of iptacopan in preventing the symptoms of aHUS over a 12-month period. Participants will be monitored to see if they remain free from signs of a condition called Thrombotic Microangiopathy (TMA), which is a complication of aHUS that affects small blood vessels. The study will involve regular check-ups and blood tests to assess the health of the participants' blood and kidneys.

Throughout the study, the health of participants will be closely observed, including any changes in blood and kidney function. The study will also track any side effects or serious health events that may occur. The goal is to determine if iptacopan can be a safe and effective alternative to the current anti-C5 antibody treatments for managing 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
Initiation of iptacopan treatment
The study begins with the switch from anti-C5 antibody therapy to iptacopan therapy. Participants will start taking iptacopan, which is administered orally in the form of hard gelatin capsules.
Before starting iptacopan, ensure vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae are up to date. If vaccinations are not completed at least two weeks prior, prophylactic antibiotics may be required.
Step 2
Treatment period
Participants will continue taking iptacopan for a duration of 12 months. The dosage and frequency will be determined based on individual needs and medical guidance.
Regular monitoring through laboratory blood tests will be conducted to assess the absence of aHUS disease and to evaluate blood and kidney health.
Step 3
Monitoring and evaluation
Throughout the 12-month treatment period, the absence of TMA (thrombotic microangiopathy) manifestation will be monitored without the use of anti-C5 antibody.
Changes in hematologic parameters such as platelet count, LDH (lactate dehydrogenase), hemoglobin, and kidney function will be assessed at the end of the treatment period.
Step 4
Safety assessments
Safety evaluations will be conducted at the end of the 12-month period. This includes monitoring for any adverse events or serious adverse events, as well as checking safety laboratory parameters and vital signs.
The requirement for dialysis and any TMA-related events will also be assessed.

Who can join the trial?

9 criteria

You must sign a form to show you understand and agree to join the study.
You need to be a man or woman who is at least 18 years old.
You should be willing and able to attend all the study visits as scheduled.
You must have been diagnosed with Atypical Hemolytic Uremic Syndrome (aHUS), and other similar conditions must have been ruled out.
You should currently be on a specific treatment called anti-C5 antibody treatment (like eculizumab or ravulizumab) for at least 3 months before the study starts.
You need to show improvement from the anti-C5 antibody treatment for at least 3 months, which means:
- Your platelet count should be at least 150 x 10⁹/L, and your LDH (a blood test) should be normal.
- Your kidney function should be stable or getting better, with no more than a 15% increase in serum creatinine (a kidney function test).
- You must be vaccinated against certain infections like Neisseria meningitidis and Streptococcus pneumoniae before starting the new treatment. If you haven't been vaccinated or need a booster, it should be done at least 2 weeks before starting the new treatment. If treatment needs to start sooner, you must take antibiotics for at least 2 weeks after vaccination.
- If you haven't been vaccinated before or need a booster, you should also get vaccinated against Haemophilus influenzae at least 2 weeks before starting the new treatment, if available.

Who cannot join the trial?

8 criteria

Patients who have any other serious medical conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who are pregnant or breastfeeding.
Patients who have a history of drug or alcohol abuse.
Patients who have an allergy or sensitivity to the study medication or its ingredients.
Patients who are unable to follow the study procedures or attend the required visits.
Patients who have a condition called **Atypical Hemolytic Uremic Syndrome (aHUS)**, which is a rare disease affecting the blood and kidneys.

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Verified sites

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Centre Hospitalier Regional Universitaire De Tours
Verified
Tours, France
Centre Hospitalier Universitaire De Bordeaux
Verified
Bordeaux, France
Fundacio De Recerca Clinic Barcelona-Institut D’Investigacions Biomediques August Pi I Sunyer
Responsive
Barcelona, Spain

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Investigated drugs

Iptacopan is a medication being studied for its effectiveness and safety in treating patients with atypical Hemolytic Uremic Syndrome (aHUS). This study focuses on patients who are switching from another treatment, specifically an anti-C5 antibody therapy, to iptacopan. The goal is to see if iptacopan can help prevent the symptoms of a condition called Thrombotic Microangiopathy (TMA) over a 12-month period.

Iptacopan

What is already known about the treatment

Iptacopan – Iptacopan is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness and safety in treating Atypical Hemolytic Uremic Syndrome (aHUS). The main therapeutic indication for iptacopan is to prevent the manifestation of thrombotic microangiopathy (TMA) in patients with aHUS. At the molecular level, iptacopan works by inhibiting a specific protein in the complement system, which is part of the immune system, thereby reducing inflammation and preventing blood vessel damage. It is classified pharmacologically as a complement inhibitor.

Investigated diseases

Atypical Hemolytic Uremic Syndrome (aHUS) – Atypical Hemolytic Uremic Syndrome is a rare disease characterized by the formation of small blood clots in the blood vessels, which can lead to damage in various organs, particularly the kidneys. It is caused by abnormal activation of the immune system, leading to the destruction of red blood cells, low platelet count, and kidney failure. The disease can progress with symptoms such as fatigue, high blood pressure, and reduced urine output. Over time, the continuous formation of clots can cause significant damage to the kidneys and other organs. The condition is chronic and can have periods of remission and relapse. It requires careful monitoring of blood and kidney health to manage its progression.