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Oral Propanolol for Surgically Inaccessible Cavernous Malformations

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ClinicalTrials.gov Identifier: NCT03523650
Recruitment Status : Enrolling by invitation
First Posted : May 14, 2018
Last Update Posted : May 14, 2018
Sponsor:
Information provided by (Responsible Party):
Yashar Kalani, MD, University of Virginia

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 Type : Interventional  (Clinical Trial)
Estimated 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 : February 7, 2018
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2019

Resource links provided by the National Library of Medicine


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.




Primary Outcome Measures :
  1. Number of symptomatic and silent hemorrhages on MRI [ Time Frame: 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 [ Time Frame: 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.



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Ages Eligible for Study:   1 Year to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03523650


Locations
United States, Virginia
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

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     History of Changes
Other Study ID Numbers: 20195
First Posted: May 14, 2018    Key Record Dates
Last Update Posted: May 14, 2018
Last Verified: May 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:
Congenital Abnormalities
Hemangioma, Cavernous, Central Nervous System
Hemangioma, Cavernous
Central Nervous System Vascular Malformations
Nervous System Malformations
Vascular Malformations
Cardiovascular Abnormalities
Propranolol
Hemangioma
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Neoplasms
Nervous System Diseases
Cardiovascular Diseases
Hemostatic Disorders
Vascular Diseases
Hemorrhagic Disorders
Hematologic Diseases
Adrenergic beta-Antagonists
Adrenergic Antagonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Anti-Arrhythmia Agents
Antihypertensive Agents
Vasodilator Agents