View Clinical Trial (Medical Research Study)


Treatment of Port-wine Mark in Sturge-Weber Syndrome Using Topical Timolol

Signup
Browse Studies

City:   Philadelphia
State:   Pennsylvania
Zip Code:   19107
Conditions:   Sturge Weber Syndrome - Port-wine Mark
Purpose:   Primary Objective: • To assess the possible utility of topical timolol in the management of port-wine mark (PWM) in Sturge-Weber syndrome in children.
Study Summary:   Port-wine mark (PWM) represents a congenital capillary malformation,characterized by dilation and malformation of dermal capillaries that lack endothelial proliferation. It is frequently seen in the facial distribution of the trigeminal nerve. PWM persists throughout life and involves ~0.3% of the population. Although PWMs are found in other circumstances, ~ 3% of patients with facial PWM are also afflicted with Sturge-Weber syndrome. PWMs are cosmetic entities that often have serious social consequences, producing psychological trauma to both children and their parents. PWM does not involute with time, and, if left untreated, can develop deep purple coloration, tissue hypertrophy, and nodularity. Laser therapy, which selectively destroys specific targets within the skin, is currently the most commonly used approach for treating PWM, although complete blanching of the PWM after laser is rarely achieved for most patients, and only 10-45% of patients with Sturge-Weber have shown satisfactory outcomes. Complications of pulsed dye laser treatment for PWM include pyogenic granuloma, scabbing, cutaneous scarring, and permanent hypo/hyperpigmentation. Laser treatment is relatively contraindicated in children with darker skin coloration due to the resulting hypopigmentation which may be equally unsightly. Laser treatment causes substantial discomfort and pain to patients, and often requires general anesthesia in children. This is particularly true since earlier treatment in infancy is desirable and yields increased successful resolution of the PWM. The hypertrophic PWM in later years is resistant to any treatment. Recently, propranolol was reported to successfully treat capillary hemangioma in infants.13 While the mechanism by which beta blockade improves hemangioma is unclear, ß2-mediated vasoconstrictive effects and the ensuing apoptosis of capillary endothelial cells may contribute to the positive therapeutic results. Oral application of propranolol can cause severe systemic complications, including bronchospasm, vasospasm, hypoglycemia, hypotension, severe bradycardia, heart block, and congestive heart failure. Topical timolol solution, a β-blocker, has shown a similar ability to reduce capillary hemangioma of eyelids with little or no systemic effects in a small pilot study. Similar to capillary hemangioma, which is a proliferative lesion characterized by increased endothelial cell turnover, PWM is a capillary malformation with abnormal endothelial cells and large surface area of dilated capillaries. Thus, both capillary hemangioma and PWM share the similar characteristic of abnormal capillary endothelial cells. This pilot study is designed to explore the potential role of topical timolol in the management of PWM. As PWM is so frequently associated with Sturge-Weber syndrome, a disorder in which approximately 50% of patients will develop glaucoma, this study will be conducted in an ophthalmology setting. This study will consist of two arms. One group will receive timolol and the second group a placebo preservative free artificial tear gel. The groups will be divided with a ratio of 1:1 and the Timolol group will be matched with the placebo group by PWM location, age and race. Both medications are to be applied and rubbed in by fingertip to the treatment site twice a day for 6 months by subject's parents/guardian. (Treatment site: 1x1 cm at inferior edge of facial PWM) Follow-up schedule: 1 week after treatment initiation and then every 2 months for a period of six months.
Criteria:   Inclusion criteria: - Age from 2 years to 10 years - Port-Wine Mark - English fluent and literate substitute decision maker - Substitute decision maker vision sufficient to read informed consent document Exclusion criteria: - Active ocular infection (conjunctivitis, keratitis,) - History of systemic conditions including hypo/hypertension, hypoglycemia, bradycardia, asthma or any contraindication to beta blocker use - Unable to comply with required follow-up - Substitute decision maker not English fluent or not literate - Substitute decision maker unable to read consent document - Patient already using systemic beta-blocker or beta-agonist (Patients already using topical beta-blocker for glaucoma will not be excluded from study).
NCT ID:   NCT01533376
Primary Contact:   Principal Investigator
Alex V Levin, MD, MHSc
Wills Eye Institute

Waleed K Abed Alnabi, MD
Phone: 2159283418
Email: Wabedalnabi@willseye.org
Backup Contact:   Email: alevin@willseye.org
Alex V Levin, MD, MHSc
Phone: 2159283918
Location Contact:   Philadelphia, Pennsylvania 19107
United States

Waleed K Abed Alnabi, MD
Phone: 215-928-3418
Email: Wabedalnabi@willseye.org

Site Status: Recruiting

Data Source:   ClinicalTrials.gov
Date Processed:   May 04, 2016
Modifications to this listing:   Only selected fields are shown, please use the link below to view all information about this clinical trial.
Click to view Full Listing


If you would like to be contacted by the clinical trial representative please enter your contact information, then click I Am Interested In This Study

Full Name:
 
Email Address:
Confirm Email:
Daytime Phone (eg. 555-555-5555):
City:
State:
Zip Code:
Best Time to Call:
Questions/Comments:
  • NEARBY STUDIES

    (updated 12 minutes ago)
Within 25 Miles

Sleep Apnea - Philadelphia PA

Alzheimer's Disease - Berlin NJ

Alzheimer's Disease - Media PA

Early Alzheimer's Disease - Jenkintown PA

Attention Deficit Hyperactivity Disorder (ADHD) - Cherry Hill NJ

Multiple Sclerosis - Willow Grove PA

Hepatitis B - Philadelphia PA

Spinal Cord Injury - Philadelphia PA

Spinal Cord Injury - Philadelphia PA

Bipolar Depression - Cherry Hill NJ

Non-Small Cell Lung Cancer (NSCLC) - Philadelphia PA

Attention Deficit Hyperactivity Disorder (ADHD) - Cherry Hill NJ

Schizophrenia (fMRI) - Philadelphia PA

Atopic Dermatitis - Berlin NJ

Liver Disease - Berlin NJ

Type 2 Diabetes - Berlin NJ

Acne Vulgaris - Berlin NJ

Post Shingles Pain - Levittown PA

Kidney Disease - Berlin NJ

Opioid Dependence - Berlin NJ

Schizophrenia - Berlin NJ

Alzheimer's disease - Berlin NJ

Healthy Adults - Berlin NJ

Major Depressive Disorder - Berlin NJ

Treatment Resistant Depression - Cherry Hill NJ

Low Back Pain - Marlton NJ

Osteoarthritis - Philadelphia PA

Mild to Moderate Alzheimer's Disease - Norristown PA

Within 50 Miles

Alzheimer's Disease - Manchester Township NJ

Alzheimer's Disease - Toms River NJ

Early Alzheimer's Disease - Lawrence Township NJ

Early Alzheimer's Disease - Manchester Township NJ

Pediatric Depression - Princeton NJ

Osteoarthritis - Downingtown PA

Rheumatoid Arthritis - Toms River NJ

Migraines - Toms River NJ

Yeast/Bacterial Vaginosis (BV) Vaginal Infections - Plainsboro NJ

Attention Deficit Hyperactivity Disorder (ADHD) - Princeton NJ

Menopausal Vaginal Dryness/Pain - Plainsboro NJ

Migraines - Allentown PA

Menopause Vaginal Laser Therapy - Plainsboro NJ

Mild to Moderate Alzheimer's Disease - Lawrence Township NJ

Mild to Moderate Alzheimer's Disease - Manchester Township NJ

Within 100 Miles

Sleep Apnea - New York NY

Alzheimer's Disease - West Long Branch NJ

Alzheimer's Disease - Brooklyn NY

Alzheimer's Disease - New Hyde Park NY

Alzheimer's Disease - New York NY

Alzheimer's Disease - New York NY

Alzheimer's Disease - Wilkes Barre PA

Early Alzheimer's Disease - Wilkes Barre PA

Depression - New York NY

Premature Ejaculation - New York NY

Arthritis (Rheumatoid Arthritis) - Baltimore MD

Polyarticular Juvenile Idiopathic Arthritis (PJIA) - Baltimore MD

Healthy Volunteers (Age 18-55) - Baltimore MD

Healthy Volunteers (Age 18-55) - Baltimore MD

Pediatric Depression - New York NY

Pediatric Depression - Staten Island NY

Pediatric Depression - Pikesville MD

Osteoarthritis (OA) of the Hip or Knee - New York NY

Low Back Pain - New York NY

Depression - New York NY

Temporomandibular Disorder (TMJD) - Baltimore MD

Postherpetic Neuralgia - New York NY

Social Anxiety Disorder - New York NY

Adult ADHD - New York NY

Depression - New York NY

Treatment-Resistant Major Depressive Disorder - New York NY

Memory Loss - New York NY

Depression - New York NY

Spinal Cord Injury - Baltimore MD

Healthy NON-Smokers - Catonsville MD

Adult ADHD - New York NY

Major Depressive Disorder - New York NY

Healthy Female Volunteers (Age 18-50) - Baltimore MD

Cat Allergies - Baltimore MD

Healthy Male Volunteers (Age 18-50) - Baltimore MD

Rheumatoid Arthritis - Reading PA

Osteoarthritis - New York NY

Diabetes and Hypertension - New York NY

Healthy Volunteers (Age 18-45) - Baltimore MD

Metastatic Pancreatic Cancer - New York NY

Metastatic Pancreatic Cancer - Bronx NY

Metastatic Pancreatic Cancer - Towson MD

Ulcerative Colitis - Great Neck NY

Arthritis (Rheumatoid Arthritis) - Baltimore MD

Non-Small Cell Lung Cancer (NSCLC) - Hershey PA

Metastatic Pancreatic Cancer - Hershey PA

Healthy Volunteers (Age 18-50) - Baltimore MD

Non-Small Cell Lung Cancer (NSCLC) - Bronx NY

Attention Deficit Hyperactivity Disorder (ADHD) - New York NY

Osteoarthritis - Brooklyn NY

Healthy Volunteers (Age 18-35) - Baltimore MD

Diabetes Type 2 - Secaucus NJ

Healthy Volunteers (Age 18-55) - Baltimore MD

Healthy Volunteers (Age 18-45) - Baltimore MD

Severe Asthma - Baltimore MD

Healthy Volunteers - Baltimore MD

Healthy Volunteers (Age 25-80) - Baltimore MD

Post Shingles Pain - New York NY

Post Shingles Pain - Brooklyn NY

Osteoarthritis - Jamaica NY

Lung Cancer - Nanuet NY

Migraines - Martinsville NJ

Migraines - Brooklyn NY

Migraines - Pikesville MD

COPD (Chronic Obstructive Pulmonary Disease) - New York NY

Cholesterol - New York NY

Constipation - Irritable Bowel Syndrome (IBS) - New York NY

Bipolar Disorder - New York NY

Healthy Female Volunteers (Age 18-70) - Baltimore MD

Seborrheic Keratosis - New York NY

Healthy Volunteers (Age 18-45) - Baltimore MD

Mild to Moderate Alzheimer's Disease - Staten Island NY