Ongoing Clinical Trials for Von Hippel-Lindau Disease
There are currently 2 ongoing clinical trials investigating new treatments for Von Hippel-Lindau disease, a rare genetic condition that causes tumors to develop in various parts of the body. These trials are being conducted in France and Denmark, testing medications that may help control tumor growth in the brain, spinal cord, and kidneys.
Clinical trial locations
- Denmark
- France
Study of Propranolol Treatment for Brain Hemangioblastomas in Patients with Von Hippel-Lindau Disease
This trial is investigating whether propranolol, a medication commonly used to treat high blood pressure, can help control the growth of hemangioblastomas in the brain and spinal cord. Hemangioblastomas are blood vessel tumors that commonly develop in people with this condition.
Who can participate:
- Adults aged 18 years and older who have been diagnosed with Von Hippel-Lindau disease
- Patients who have at least one hemangioblastoma in the brain or spinal cord
- The tumor must not require immediate surgery within the next 3 months
- Both men and women can participate
- Patients must have active health insurance coverage
Who cannot participate:
- People with severe allergic reactions to propranolol or similar medications
- Patients with slow heart rate (below 60 beats per minute at rest)
- Those with low blood pressure (systolic pressure below 100 mmHg)
- People with severe asthma or chronic breathing problems
- Patients with heart failure or significant heart problems
- Pregnant or breastfeeding women
- Those currently using other beta-blocker medications
- Patients with severe kidney or liver problems, uncontrolled diabetes, or depression
What the trial involves: Participants will be divided into two groups. One group will receive propranolol tablets, and the other will receive a placebo. The treatment continues for 24 months, with patients taking the medication orally every day, up to a maximum dose of 120 mg. Regular check-ups every 6 months will include MRI scans to monitor tumor size and growth rate. Blood samples will be collected to measure proteins related to tumor growth, and doctors will assess how patients are feeling and functioning in their daily activities.
Investigational drug: Propranolol is a beta-blocker that works by blocking certain natural chemicals in the body that affect the heart and blood vessels. While commonly used for high blood pressure and heart conditions, researchers are investigating whether it can help control the growth of blood vessel tumors in the brain and spinal cord.
Study on Belzutifan for Treating Kidney Cancer in Patients with von Hippel-Lindau Disease
This trial is testing a medication called Belzutifan (also known as MK-6482 or PT2977) for treating kidney cancer that develops in people with Von Hippel-Lindau disease. The medication is taken orally as a film-coated tablet.
Who can participate:
- Adults aged 18 years and older with a confirmed diagnosis of Von Hippel-Lindau disease
- Patients who have at least one measurable kidney cancer tumor
- No kidney cancer tumor larger than 3.0 cm that needs immediate surgery
- Patients must be fully active or have some symptoms but not need to stay in bed (ECOG performance status 0 to 1)
- Must have proper organ and bone marrow function
- Must be able to swallow oral medications and follow study procedures
- Both men and women can participate, but must agree to use contraception during the study
- Women must not be pregnant or breastfeeding and must have a negative pregnancy test before starting
Who cannot participate:
- Patients who do not have Von Hippel-Lindau disease-associated kidney cancer
- Those who are not within the specified age range
- Vulnerable populations who may need special protection in research
What the trial involves: After providing informed consent and confirming eligibility, patients will begin taking Belzutifan tablets orally. The dosage and frequency will be determined by the study protocol. Regular monitoring will assess how the tumors respond to treatment, including measuring tumor size and progression. The study will evaluate the overall response rate, how long the response lasts, and progression-free survival. The study is estimated to conclude by December 30, 2026.
Investigational drug: Belzutifan is a targeted cancer therapy that works by inhibiting a protein called hypoxia-inducible factor-2 alpha (HIF-2α), which plays a role in tumor growth and survival. This medication is being studied to see how well it can reduce the size of kidney tumors or stop them from growing.
Summary
Both ongoing clinical trials for Von Hippel-Lindau disease focus on different aspects of the condition. One trial is testing propranolol for brain and spinal cord tumors, while the other is investigating Belzutifan for kidney cancer. France is conducting both trials, making it the primary location for this research, while Denmark is also participating in the kidney cancer study. Interestingly, the propranolol trial represents a potential repurposing of an established medication for a new use, while Belzutifan is a newer targeted therapy specifically designed to work against the mechanisms of tumor growth in this genetic condition.
