Actively Recruiting
Propranolol and Von Hippel-Lindau Disease
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2023-12-13
85
Participants Needed
1
Research Sites
197 weeks
Total Duration
On this page
Sponsors
A
Assistance Publique - Hôpitaux de Paris
Lead Sponsor
A
Agence Générale des Equipements et Produits de Santé
Collaborating Sponsor
AI-Summary
What this Trial Is About
Propranolol (beta-blocker), is successfully used for the treatment of infantile hemangiomas, the most common vascular tumor of newborns. The mechanism is related to its anti-angiogenetic and pro-apoptotic effects. Recently, in vitro studies demonstrated that propranolol decreased the expression of target genes of the HIF (hypoxia-inducible factor, of which the VHL gene is the main regulator) pathway in hemangioblastoma cells and affected their viability. The efficacy of propranolol (stabilization of all HB and decrease in serum VEGF levels) was demonstrated in a phase III study, but only in retinal BHs . The only study that evaluated the effect of propranolol on CNS HB was retrospective and involved a limited number of patients. Nevertheless, it showed a decrease in the growth rate of HBs. The investigator therefore propose to carry out a randomized controlled trial to study the effect of propranolol on the growth of CNS HB in patients with VHL disease (von Hippel-Lindau). The hypothesis of the present work is the following: the use of propranolol in VHL patients with CNS HB allows to decrease and/or slow down the tumor growth.
CONDITIONS
Official Title
Propranolol and Von Hippel-Lindau Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosed with Von Hippel-Lindau disease with one or more central nervous system hemangioblastomas not requiring urgent surgery within 3 months
- Provided written consent to participate
- Enrolled in a social security plan or beneficiary
You will not qualify if you...
- Contraindications to propranolol including chronic obstructive pulmonary disease, asthma, uncontrolled heart failure, 2nd or 3rd degree atrioventricular blocks, bradycardia below 50 beats per minute after 3 minutes rest, Raynaud's phenomenon, 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
- Advanced liver failure with hyperbilirubinemia, massive ascites, hepatic encephalopathy
- Predisposition to hypoglycemia or metabolic acidosis
- Contraindications to MRI such as claustrophobia, pacemaker or implants, ocular metallic foreign bodies, heart valves or ferromagnetic metal vascular clips
- Current use of propranolol or other beta blockers
- Under guardianship or conservatorship
- Pregnant or breastfeeding women or women with medium-term pregnancy plans
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
AP-HP, Bicêtre Hospital
Le Kremlin-Bicêtre, France, 94275
Actively Recruiting
Research Team
A
AGHAKHANI Nozar, PR
CONTACT
R
RICHARD Stéphane, PR
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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