Using Propranolol to Treat Hemangioblastomas in Von Hippel-Lindau Disease
Efficacy of Propranolol for the Treatment of Central Nervous System Hemangioblastomas in Von Hippel-Lindau Disease: a Randomized Controlled Clinical Trial
This study is testing if the medication propranolol can help people with Von Hippel-Lindau disease by slowing the growth of brain tumors called hemangioblastomas without needing surgery.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 85 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 1 site (Le Kremlin-Bicêtre) |
| Trial ID | NCT05424016 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effects of propranolol, a beta-blocker known for its anti-angiogenetic properties, on the growth of central nervous system hemangioblastomas in patients with Von Hippel-Lindau disease. The study aims to determine whether propranolol can stabilize hemangioblastomas and reduce serum VEGF levels, potentially offering a non-surgical treatment option. Participants will be randomized to receive propranolol or follow-up care, with careful monitoring of tumor growth and patient health. The trial is designed to provide more robust evidence compared to previous studies that had limited patient numbers.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older diagnosed with Von Hippel-Lindau disease and have one or more central nervous system hemangioblastomas that do not require urgent surgery.
Not a fit: Patients with contraindications to propranolol, such as severe respiratory or cardiac conditions, will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a non-invasive option to manage hemangioblastomas, reducing the need for surgical interventions and associated complications.
How similar studies have performed: Previous studies have shown some success with propranolol in treating retinal hemangioblastomas, but this approach for CNS hemangioblastomas is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age ≥ 18 * VHL patient with one or more hemangioblastomas of the central nervous system, none of which require urgent surgery (within 3 months) * Patient with written consent to participate in the study * Enrolled in a social security plan or beneficiary Exclusion Criteria: * Contraindication to the use of propranolol: * chronic obstructive pulmonary disease and asthma, * uncontrolled heart failure, * 2nd and 3rd degree atrioventricular blocks, * bradycardia (\<50 beats/minute after 3 minutes of rest), * Raynaud's phenomenon and peripheral arterial disorders, * arterial hypotension, * hypersensitivity to propranolol * cardiogenic shock, * Prinzmetal's angina, * sinus disease (including sino-auricular block) * untreated pheochromocytoma, * history of anaphylactic reaction, * in the context of primary and secondary prevention of digestive bleeding in cirrhotics: advanced liver failure with hyperbilirubinemia, massive ascites, hepatic encephalopathy * predisposition to hypoglycemia (as after fasting or in case of abnormal response to hypoglycemia) * metabolic acidosis * Contraindication to MRI: * claustrophobia, * presence of a pace maker and other stimulators/implants * ocular metallic foreign bodies, * heart valves or ferromagnetic metal vascular clips * Patients already on Propranolol or other beta blockers * Patients under guardianship or conservatorship * Pregnant or breastfeeding women - Woman with a medium-term pregnancy project
Where this trial is running
Le Kremlin-Bicêtre
- AP-HP, Bicêtre Hospital — Le Kremlin-Bicêtre, France (Recruiting)
Study contacts
- Study coordinator: AGHAKHANI Nozar, PR
- Email: nozar.aghakhani@aphp.fr
- Phone: +33145212380
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.