Oral Propanolol for Surgically Inaccessible Cavernous Malformations

• Sponsor: University of Virginia (Other)
• Overall Status: Unknown status
• CT.gov ID: NCT03523650
• Collaborator: (none)
• Enrollment: 346
• Location: 1
• Arms: 2
• Anticipated Duration (Months): 10.8
• Patients Per Site Per Month: 32.1

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Cavernous Malformations,Cerebral and/or Spinal	Drug: Propranolol Oral Tablet; Drug: Placebo Oral Tablet	Phase 1

Detailed 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.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 346 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Oral Propanolol for Surgically Inaccessible Cerebral and Spinal Cavernous Malformations
• Actual Study Start Date: Feb 7, 2018
• Anticipated Primary Completion Date: Jan 1, 2019
• Anticipated Study Completion Date: Jan 1, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Group 1: Propranolol Group Group 1: Propranolol - group of randomized patients will receive one propranolol pill tid for 36 months.	Drug: Propranolol Oral Tablet; A randomized group of patients with cerebral or spinal cavernous malformations, will receive a Propranolol Oral tablets, tid, for 36 months.
Placebo Comparator: Group 2: Placebo Group Group 2: Placebo - group of randomized patients will receive one placebo pill tid for 36 months.	Drug: Placebo Oral Tablet; A randomized group of patients with cerebral or spinal cavernous malformations, will receive a Placebo Oral Tablet, tid, for 36 months.

Outcome Measures

Primary Outcome Measures

1. Number of symptomatic and silent hemorrhages on MRI [36 months]

   Using MRI imaging the size of the cavernous malformation will be measured. The primary outcome is the number of symptomatic and silent hemorrhages as assessed by review of a fine-cut, axial T2-weighted MRI obtained at each visit.

Secondary Outcome Measures

1. Rate of de novo lesion formation; changes in rate of breakthrough seizures or other neurological deficits [36 months]

   The secondary outcome is the rate of de novo lesion formation; changes in rate of breakthrough seizures or other neurological deficits; quality of life measured using the SF-36 at each visit, patient satisfaction with treatment; and the incidence of treatment failure.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 1 Year to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

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.

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

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

3. 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Univeristy of Virginia	Charlottesville	Virginia	United States	22903

Sponsors and Collaborators

• University of Virginia

Investigators

• Principal Investigator: Yashar Kalani, MD, PHD, University of Virginia Medical Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Yashar Kalani, MD, Co-Director of Neurovascular Surgery, Director of Skull Base Surgy and Director of Stroke Center, University of Virginia
• ClinicalTrials.gov Identifier: NCT03523650
• Other Study ID Numbers: 20195
• First Posted: May 14, 2018
• Last Update Posted: May 14, 2018
• Last Verified: May 1, 2018

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes
• Additional relevant MeSH terms: Hemangioma, Cavernous, Central Nervous System; Congenital Abnormalities; Propranolol

Study Results

No Results Posted as of May 14, 2018