Ongoing Clinical Trials for Hereditary Haemorrhagic Telangiectasia
There is currently 1 ongoing clinical trial investigating new treatment options for hereditary haemorrhagic telangiectasia, a genetic disorder affecting blood vessels that can cause frequent nosebleeds and other complications. This trial is exploring whether nintedanib, a medication that blocks proteins involved in blood vessel growth, can effectively reduce the duration of nosebleeds in patients with this condition. (Also known as: HHT, Osler-Weber-Rendu syndrome, Rendu-Osler-Weber disease)
Clinical trial locations
Study on the Effectiveness of Nintedanib for Treating Nosebleeds in Patients with Hereditary Hemorrhagic Telangiectasia
This clinical trial is being conducted in France and focuses on whether nintedanib can help reduce the duration and frequency of nosebleeds in patients with hereditary haemorrhagic telangiectasia. The study involves a 16-week treatment period following an initial 8-week observation phase to establish baseline nosebleed patterns.
Main inclusion criteria:
- Adults aged 18 years or older
- Confirmed diagnosis of hereditary haemorrhagic telangiectasia through genetic testing showing a mutation in one of the HHT genes, or meeting 3 out of 4 specific clinical criteria
- Moderate to severe nosebleeds, measured by an Epistaxis Severity Score of 3 or higher
- No abnormal connections between arteries and veins in the brain, as confirmed by brain imaging
- Signed informed consent to participate in the study
Main exclusion criteria:
- History of allergic reactions to the study medication
- Currently taking other medications that might interfere with the study treatment
- Severe liver disease
- Uncontrolled high blood pressure
- Pregnancy or breastfeeding
- History of cancer in the last 5 years, with the exception of certain types of skin cancer
- Any other medical condition that study doctors determine might make participation unsafe
Focus and goal of the trial:
The primary goal of this study is to determine whether patients receiving nintedanib experience at least a 30% reduction in the duration of their nosebleeds compared to the baseline period before treatment. Participants are randomly assigned to receive either nintedanib or a placebo in the form of oral soft capsules. Throughout the 16-week treatment phase, the duration of nosebleeds is carefully monitored and compared to the initial 8-week observation period. This research aims to provide valuable information about whether nintedanib can be a viable treatment option for managing nosebleeds in patients with this condition, potentially improving quality of life for those affected.
Investigational drug:
The study uses nintedanib, also known by its code name BIBF 1120 and marketed as Ofev. This medication is administered orally in 150 mg soft capsules. Nintedanib works by blocking certain proteins in the body that are involved in the growth of blood vessels. It is classified as a tyrosine kinase inhibitor, which means it blocks specific enzymes that help new blood vessels form. By inhibiting these proteins, nintedanib may help reduce the frequency and duration of nosebleeds in patients with hereditary haemorrhagic telangiectasia.
Summary
Currently, there is one active clinical trial investigating treatment options for hereditary haemorrhagic telangiectasia. This trial is being conducted in France and specifically targets one of the most common and troublesome symptoms of the condition: frequent nosebleeds. The study focuses on nintedanib, a tyrosine kinase inhibitor that blocks proteins involved in blood vessel formation, to determine if it can significantly reduce the duration of nosebleeds in affected patients.
The trial uses a randomized, placebo-controlled design over a 16-week treatment period, preceded by an 8-week baseline observation phase. The study is seeking adults with confirmed diagnoses who experience moderate to severe nosebleeds. Notable exclusion criteria include severe liver disease, uncontrolled high blood pressure, and recent cancer history, which reflect the need to ensure participant safety during the trial.
This research represents an important step in exploring new treatment options for managing the bleeding complications associated with hereditary haemorrhagic telangiectasia, a condition that can significantly impact daily life and overall wellbeing.
