Study of Propranolol Treatment for Brain Hemangioblastomas in Patients with Von Hippel-Lindau Disease

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What is this study about?

This clinical trial focuses on von Hippel-Lindau disease, a rare genetic condition that causes tumors to grow in different parts of the body. Specifically, the study examines tumors called hemangioblastomas that develop in the brain and spinal cord (central nervous system). The study will test whether propranolol, a medication commonly used to treat high blood pressure, can help control the growth of these tumors.

The study will investigate how well propranolol works in treating brain and spinal cord tumors in people with von Hippel-Lindau disease. Participants will be divided into two groups, with one group receiving propranolol tablets and the other receiving a placebo. The treatment will continue for 24 months, during which patients will take the medication orally every day. The maximum daily dose of propranolol will be 120 mg.

Throughout the study, patients will have regular check-ups every 6 months, including MRI scans to monitor their tumors. Doctors will measure tumor size, growth rate, and other changes in the brain. They will also collect blood samples to measure certain proteins related to tumor growth and assess how patients are feeling and functioning in their daily activities. This will help determine if propranolol is effective in treating these tumors.

1 Initial evaluation

You will undergo initial evaluation to confirm eligibility for the study. This includes verification that you are at least 18 years old and have von Hippel-Lindau disease with one or more brain lesions that do not require immediate surgery.

Your doctor will perform baseline measurements of your brain lesions using magnetic resonance imaging (MRI).

A blood sample will be taken to measure VEGF levels (a protein related to blood vessel growth).

2 Treatment assignment

You will be randomly assigned to receive either propranolol or standard care.

The medication propranolol is taken by mouth.

3 Regular monitoring

You will have MRI scans every 6 months to monitor your brain lesions.

Your quality of life and physical abilities will be assessed every 6 months using standard questionnaires.

Blood tests will be performed at 12 months and 24 months to measure VEGF levels.

Any side effects from the treatment will be recorded during the entire study period.

4 Final evaluation

At 24 months, a final comprehensive evaluation will be conducted.

This includes an MRI scan, blood test, and assessment of your physical condition and quality of life.

Two specialists will review your MRI results to determine how your brain lesions have responded to treatment.

Who Can Join the Study?

Must be at least 18 years of age
Must have been diagnosed with Von Hippel-Lindau (VHL) disease
Must have at least one central nervous system hemangioblastoma (a type of benign blood vessel tumor in the brain or spinal cord)
The hemangioblastomas must not require immediate surgery within the next 3 months
Must be able to provide written informed consent to participate in the research
Must have active health insurance coverage or be a dependent on someone else’s health insurance plan
Both men and women can participate in this study

Who Cannot Join the Study?

History of severe allergic reactions to propranolol or similar medications (beta-blockers)
Heart rate below 60 beats per minute at rest
Low blood pressure (systolic pressure below 100 mmHg)
Severe asthma or other chronic breathing problems
History of heart failure or significant heart problems
Pregnancy or breastfeeding
Current use of other beta-blocker medications (medications that slow heart rate)
Severe kidney or liver problems
Patients with uncontrolled diabetes (as beta-blockers can mask symptoms of low blood sugar)
History of depression or other significant mental health conditions
Current participation in other clinical trials
Inability to provide informed consent
Known drug or alcohol dependency
Patients who have had major surgery within the last 3 months
People with conditions that might interfere with the study medication absorption

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Hopital Beaujon	Clichy	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	16.01.2023

Trial locations

Investigated drugs:

Propranolol

Propranolol is a beta-blocker medication that works by blocking certain natural chemicals in your body (such as epinephrine) that affect the heart and blood vessels. In this trial, it is being studied for its potential effects on treating blood vessel tumors (hemangioblastomas) that develop in the brain and spinal cord of patients with von Hippel-Lindau disease. While this medication is commonly used to treat high blood pressure, irregular heartbeats, and other heart conditions, researchers are investigating whether it can help control the growth of these specific tumors.

Investigated diseases:

Von Hippel-Lindau disease

Von Hippel-Lindau Disease – A rare genetic disorder that causes blood vessels to grow abnormally and form tumors in multiple organs. The condition primarily affects the central nervous system, creating growths called hemangioblastomas in the brain, spinal cord, and retina. These blood vessel tumors can also develop in the kidneys, pancreas, and adrenal glands. The disease typically begins to show signs in young adulthood, though it can appear at any age. The condition is inherited in an autosomal dominant pattern, meaning a person needs only one copy of the altered gene to develop the disorder.

Trial ID:

2024-516241-39-00

Protocol code:

APHP210075

NCT ID:

NCT05424016

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of Propranolol Treatment for Brain Hemangioblastomas in Patients with Von Hippel-Lindau Disease

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?