Oral Propanolol for Surgically Inaccessible Cavernous Malformations

University of Virginia

Status and phase: Unknown; Phase 1

Conditions: Cavernous Malformations,Cerebral and/or Spinal

Treatments: Drug: Propranolol Oral Tablet; Drug: Placebo Oral Tablet

Study type: Interventional

Funder types: Other

Identifiers

NCT03523650

20195

Details and patient eligibility

About

The purpose of this study is to evaluate the role of propranolol as an alternative treatment for cavernous malformation in patients that may not be ideal candidates for surgery.

Full description

Patients between the ages of 1 and 80 years of age, that have been diagnosed with a spinal or cerebral cavernous malformation(s) that can not be surgically treated, will be enrolled in this randomized study. All eligible patients will be enrolled in one of two groups. Group 1 will receive oral propranolol tablets 3 times a day for 36 months. Group 2 will receive oral placebo tablets 3 times per day for 36 months. Prior to beginning treatment, all imaging, including MRIs, will be evaluated. During the first visit a blood sample will be collected for genomic analysis. Patients will return to clinic every 6 months. Each time the patient returns to clinic monitoring of how the patient is feeling and evaluation of how the patient is tolerating the propranolol will take place. At the end of the three years, evaluators will determine if the propranolol was able to stop the growth of the cavernous malformation and change the imaging features of the lesion on MRI. Once the study is completed patients will no longer receive the study drug.

Enrollment: 346 estimated patients

Sex: All

Ages: 1 to 80 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

1. Diagnosis of isolated or familial cavernous malformation syndrome:

   • symptomatic cavernous malformation not amenable to surgical resection.
   • familial cavernous malformation with seizure, other neurological symptom or surgically inaccessible lesion.

2. Written and informed consent obtained prior to study enrollment.

3. Subject is able and willing to return for outpatient visits.

4. Negative pregnancy test at time of enrollment for women and child-bearing potential.

Exclusion criteria

1. Age less than 1 year-old.
2. Propranolol allergy or allergy to other b-blockers.
3. Estimated life expectancy of less than 1 year.
4. History of severe anemia, cardiac dysfunction, or diabetes.
5. A psychiatric or substance abuse problem that may interfere with study compliance.
6. Pregnant and lactating women.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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346 participants in 2 patient groups, including a placebo group

Group 1: Propranolol Group

Experimental group

Description:

Group 1: Propranolol - group of randomized patients will receive one propranolol pill tid for 36 months.

Treatment:

Drug: Propranolol Oral Tablet

Group 2: Placebo Group

Placebo Comparator group

Description:

Group 2: Placebo - group of randomized patients will receive one placebo pill tid for 36 months.

Treatment:

Drug: Placebo Oral Tablet