Efficacy of Propranolol for the Treatment of Central Nervous System Hemangioblastomas in Von Hippel-Lindau Disease: a Randomized Controlled Clinical Trial

Status: Recruiting
Location: See location...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
France
AP-HP, Bicêtre Hospital
RECRUITING
Le Kremlin-bicêtre
Contact Information
Primary
AGHAKHANI Nozar, PR
nozar.aghakhani@aphp.fr
+33145212380
Backup
RICHARD Stéphane, PR
stephane.richard@univesite-paris-saclay.fr
+33145217201
Time Frame
Start Date: 2023-01-16
Estimated Completion Date: 2026-11-01
Participants
Target number of participants: 85
Treatments
Experimental: Experimental arm
Patients with no contraindications will be included and randomized to receive propranolol orally for 24 months
Other: control arm
Patient included with a routine follow-up
Sponsors
Collaborators: Agence Générale des Equipements et Produits de Santé
Leads: Assistance Publique - Hôpitaux de Paris

This content was sourced from clinicaltrials.gov