Treat_CCM: Propranolol in Familial Cerebral Cavernous Malformation

• Status: Active, not recruiting
• Trial end date: 2021-12-31
• Target enrollment: 0
• Participant gender: All

Summary

Cerebral Cavernous Malformation (CCM) is a cerebrovascular disease which can be either congenital in origin or sporadic and is characterized by the presence of isolated or multiple CCM lesions, causing recurrent headache, seizures, focal neurological deficits and hemorrhages. Inasmuch, to date, the only curative treatment available is limited to surgical lesion eradication or stereotactic radiosurgery. It is therefore necessary to find an effective medical treatment that may limit disease progression and decrease the burden of adverse clinical events. The non-selective betablocker propranolol has been found to be effective in the treatment of infantile cutaneous hemangioma, and anecdotal reports have been published on its efficacy in CCM. The safety profile of propranolol has been documented in millions of patients of all ages.

The primary objective of this exploratory trial is to test whether a chronic treatment with propranolol will reduce the burden of cerebrovascular lesions, of clinical events and symptoms in patients with familial CCM.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Mario Negri Institute for Pharmacological Research
• Collaborator: IFOM, The FIRC Institute of Molecular Oncology
• Treatments: Propranolol

Criteria

Inclusion Criteria:

1. Patients with Familial cerebral cavernous malformations (FCCM);

2. history of clinical symptoms or events: intracerebral hemorrhage, stroke, permanent or transient focal deficits, seizures, disability or any other neurological symptom supposedly related to CCM;

3. age of at least 18 years.

4. Written informed consent to participate in the study prior to any study procedures.

Exclusion Criteria:

1. Implanted pacemaker or any other condition preventing the magnetic resonance imaging (MRI);

2. bradycardia (<50 bpm) or 2nd or 3rd degree AV block, hypotension (symptomatic);

3. unstable diabetes;

4. severe asthma;

5. renal and/or liver failure;

6. current use of verapamil and diltiazem for risk of excessive bradycardia;

7. previous brain surgery (within 6 months);

8. known hypersensitivity to study drug (propranolol or any of the ingredients)

9. pregnant or lactating women or women of childbearing potential who are not protected from pregnancy by an accepted method of contraception

10. participation to another clinical trial;

11. inability to cooperate with the trial procedures.