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

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What is this trial about?

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

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 2

    Treatment assignment

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

    The medication propranolol is taken by mouth.

  3. Step 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. Step 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 trial?

7 criteria

  • 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 trial?

15 criteria

  • 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
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Investigated drugs

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.

What is already known about the treatment

Propranolol – A beta-blocker medication administered orally in tablet form that has been established in medical practice for decades and is now being investigated for a new potential use in treating central nervous system hemangioblastomas in Von Hippel-Lindau disease. The drug works by blocking beta-adrenergic receptors in the body, primarily affecting the heart and blood vessels, and has traditionally been used to treat high blood pressure, irregular heart rhythms, and anxiety. In this new application, researchers are studying its potential anti-angiogenic properties that may help control the growth of blood vessel tumors in the brain and spinal cord. This medication belongs to the class of beta-adrenergic blocking agents and represents a potential repurposing of an established drug for a rare genetic condition.

Investigated diseases

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516241-39-00Protocol codeAPHP210075Estimated enrolment85 patientsSponsorAssistance Publique Hopitaux De Paris

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).