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Study on the Effectiveness of Nintedanib for Treating Nosebleeds in Patients with Hereditary Hemorrhagic Telangiectasia

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 clinical trial is focused on studying the effectiveness of a medication called nintedanib for treating a condition known as hereditary hemorrhagic telangiectasia (HHT). HHT is a genetic disorder that affects blood vessels and can lead to frequent nosebleeds, known medically as epistaxis. The study aims to determine if patients with HHT experience a significant reduction in the duration of their nosebleeds when treated with nintedanib compared to those receiving a placebo.

Participants in the study will be randomly assigned to receive either nintedanib, which is also known by its code name BIBF 1120, or a placebo. The medication is administered in the form of soft capsules taken orally. The study will last for a total of 16 weeks, during which the duration of nosebleeds will be monitored and compared to the baseline period before the treatment started. The goal is to see if there is at least a 30% reduction in the duration of nosebleeds in those taking nintedanib.

The trial is designed to provide valuable information on whether nintedanib can effectively reduce the frequency and duration of nosebleeds in patients with HHT. This could potentially lead to improved management of the condition and better quality of life for those affected. The study will help determine if nintedanib is a viable treatment option for this specific aspect of HHT.

The research process

The trial runs in 5 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 the informed consent form is signed. This confirms understanding and agreement to participate in the trial.
Eligibility is confirmed by checking for hereditary haemorrhagic telangiectasia (HHT) through a genetic test or clinical criteria, being at least 18 years old, experiencing moderate to serious nosebleeds, and having no brain abnormalities as shown by imaging.
Step 2
Baseline observation
The initial phase involves an observation period lasting 8 weeks. During this time, the average duration of nosebleeds is recorded to establish a baseline.
Step 3
Treatment phase
The treatment phase lasts for 16 weeks. Participants are randomly assigned to receive either nintedanib or a placebo.
If assigned to the nintedanib group, take Ofev 150 mg soft capsules orally as directed. The specific dosage and frequency will be provided by the study team.
Step 4
Monitoring and assessment
Throughout the treatment phase, regular monitoring is conducted to assess the duration of nosebleeds.
The primary goal is to determine if there is at least a 30% reduction in the duration of nosebleeds compared to the baseline.
Step 5
Final evaluation
At the end of the 16-week treatment period, a final evaluation is conducted to compare the average duration of nosebleeds during the last 12 weeks of treatment with the baseline data.

Who can join the trial?

5 criteria

Signed informed consent: You need to agree to participate in the study by signing a form.
Definite HHT disease: You must have a confirmed diagnosis of Hereditary Haemorrhagic Telangiectasia, either through a genetic test showing a mutation in one of the HHT genes or by meeting 3 out of 4 specific clinical criteria.
Aged 18 years or older at the time of giving consent.
Moderate to serious epistaxis: You must have nosebleeds that are considered moderate to severe, as measured by an Epistaxis Severity Score of 3 or higher.
Absence of cerebral arteriovenous malformation: You must not have abnormal connections between arteries and veins in the brain, as shown by brain imaging tests.

Who cannot join the trial?

7 criteria

Patients with a history of **allergic reactions** to the study medication cannot participate. An allergic reaction is when your body reacts badly to something, like a rash or trouble breathing.
Patients who are currently taking other medications that might interfere with the study treatment are not eligible. This means if you are on certain drugs that could affect how the study medicine works, you cannot join.
Patients with **severe liver disease** cannot participate. Severe liver disease means your liver is not working well, which can affect your overall health.
Patients with **uncontrolled high blood pressure** are excluded. This means if your blood pressure is too high and not managed by medication or lifestyle changes, you cannot join.
Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
Patients with a history of **cancer** in the last 5 years, except for certain types of skin cancer, are not eligible. Cancer is a disease where cells in the body grow uncontrollably.
Patients with any other medical condition that the study doctors think might make it unsafe for them to participate are excluded. This means if you have a health issue that could be risky during the study, you cannot join.

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Verified sites

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Verified
Centre Hospitalier Lyon Sud
Pierre Benite, France
Verified
University Hospital Of Clermont-Ferrand
Clermont Ferrand, France

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Investigated drugs

Nintedanib is a medication being studied for its potential to help reduce nosebleeds in people with a condition called hereditary hemorrhagic telangiectasia (HHT). HHT is a disorder that affects blood vessels and can cause frequent nosebleeds. Nintedanib works by blocking certain proteins in the body that are involved in the growth of blood vessels. By doing this, it may help to reduce the frequency and duration of nosebleeds in patients with HHT. The goal of the study is to see if patients taking nintedanib experience a significant reduction in the duration of their nosebleeds compared to those who do not take the medication.

Nintedanib

What is already known about the treatment

Nintedanib – Nintedanib is administered orally in the form of capsules. It is currently being studied in clinical trials for its effectiveness in treating nosebleeds in patients with hereditary hemorrhagic telangiectasia (HHT), a genetic disorder that affects blood vessels. The main therapeutic indication being investigated is the reduction of nosebleed duration in HHT patients. At the molecular level, nintedanib works by inhibiting certain enzymes that are involved in the formation of new blood vessels, which may help reduce bleeding. It is classified as a tyrosine kinase inhibitor, a type of medication that blocks specific proteins involved in the growth of cancer cells and blood vessels.

Investigated diseases

Hereditary hemorrhagic telangiectasia – This is a genetic disorder that affects blood vessels, leading to abnormal connections between arteries and veins. These connections, known as telangiectasias, can cause bleeding, particularly in the nose, leading to frequent nosebleeds. Over time, these abnormal blood vessels can also form in other parts of the body, such as the lungs, liver, and brain. As the disease progresses, it can result in complications due to bleeding or the formation of arteriovenous malformations. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder. Symptoms can vary widely among individuals, even within the same family.