Treatment of Port-wine Mark in Sturge-Weber Syndrome Using Topical Timolol
NCT ID: NCT01533376
Last Updated: 2019-03-21
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE1
3 participants
INTERVENTIONAL
2012-02-29
2019-02-28
Brief Summary
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• To assess the possible utility of topical timolol in the management of port-wine mark (PWM) in Sturge-Weber syndrome in children.
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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Timolol
Participants in this group will receive topical timolol
Timolol
0.5% timolol maleate ophthalmic gel-forming solution applied once
Placebo
Participants in this group will receive Preservative free artificial tear gel.
Preservative free artificial tear gel.
Preservative free artificial tear gel applied topically twice a day.
Interventions
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Timolol
0.5% timolol maleate ophthalmic gel-forming solution applied once
Preservative free artificial tear gel.
Preservative free artificial tear gel applied topically twice a day.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Port-Wine Mark
* English fluent and literate substitute decision maker
* Substitute decision maker vision sufficient to read informed consent document
Exclusion Criteria
* 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).
2 Years
10 Years
ALL
No
Sponsors
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University of Medicine and Dentistry of New Jersey
OTHER
Wills Eye
OTHER
Responsible Party
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Dr. Alex V. Levin, MD, MHSc
Chief, Pediatric Ophthalmology and Ocular Genetics
Principal Investigators
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Alex V Levin, MD, MHSc
Role: PRINCIPAL_INVESTIGATOR
Wills Eye Institute
Locations
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Wills Eye Institute
Philadelphia, Pennsylvania, United States
Countries
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References
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Hennedige AA, Quaba AA, Al-Nakib K. Sturge-Weber syndrome and dermatomal facial port-wine stains: incidence, association with glaucoma, and pulsed tunable dye laser treatment effectiveness. Plast Reconstr Surg. 2008 Apr;121(4):1173-1180. doi: 10.1097/01.prs.0000304606.33897.71.
Jia W, Sun V, Tran N, Choi B, Liu SW, Mihm MC Jr, Phung TL, Nelson JS. Long-term blood vessel removal with combined laser and topical rapamycin antiangiogenic therapy: implications for effective port wine stain treatment. Lasers Surg Med. 2010 Feb;42(2):105-12. doi: 10.1002/lsm.20890.
Chiummariello S, Mezzana P, Fioramonti P, Onesti MG, Alfano C, Scuderi N. The use of laser and Varioscope in the management of hemangiomas and vascular malformations. Acta Chir Plast. 2006;48(1):20-5.
Li W, Yamada I, Masumoto K, Ueda Y, Hashimoto K. Photodynamic therapy with intradermal administration of 5-aminolevulinic acid for port-wine stains. J Dermatolog Treat. 2010 Jul;21(4):232-9. doi: 10.3109/09546630903159099.
Kautz G, Kautz I, Segal J, Zehren S. Treatment of resistant port wine stains (PWS) with pulsed dye laser and non-contact vacuum: a pilot study. Lasers Med Sci. 2010 Jul;25(4):525-9. doi: 10.1007/s10103-009-0727-7. Epub 2009 Dec 15.
Maruani A. [Sturge-Weber syndrome]. Presse Med. 2010 Apr;39(4):482-6. doi: 10.1016/j.lpm.2009.07.030. Epub 2010 Mar 10. French.
Onesti MG, Fioramonti P, Carella S, Spinelli G, Scuderi N. Surgical and laser treatment of Sturge-Weber syndrome. Aesthetic Plast Surg. 2009 Jul;33(4):666-8. doi: 10.1007/s00266-009-9327-y. Epub 2009 Mar 19.
Latkowski IT, Wysocki MS, Siewiera IP. [Own clinical experience in treatment of port-wine stain with KTP 532 nm laser]. Wiad Lek. 2005;58(7-8):391-6. Polish.
Li G, Lin T, Wu Q, Zhou Z, Gold MH. Clinical analysis of port wine stains treated by intense pulsed light. J Cosmet Laser Ther. 2010 Feb;12(1):2-6. doi: 10.3109/14764170903449778.
Wareham WJ, Cole RP, Royston SL, Wright PA. Adverse effects reported in pulsed dye laser treatment for port wine stains. Lasers Med Sci. 2009 Mar;24(2):241-6. doi: 10.1007/s10103-008-0560-4. Epub 2008 Apr 17.
Minkis K, Geronemus RG, Hale EK. Port wine stain progression: a potential consequence of delayed and inadequate treatment? Lasers Surg Med. 2009 Aug;41(6):423-6. doi: 10.1002/lsm.20788.
Izikson L, Nelson JS, Anderson RR. Treatment of hypertrophic and resistant port wine stains with a 755 nm laser: a case series of 20 patients. Lasers Surg Med. 2009 Aug;41(6):427-32. doi: 10.1002/lsm.20793.
Leaute-Labreze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taieb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. No abstract available.
Siegfried EC, Keenan WJ, Al-Jureidini S. More on propranolol for hemangiomas of infancy. N Engl J Med. 2008 Dec 25;359(26):2846; author reply 2846-7. doi: 10.1056/NEJMc086443. No abstract available.
Guo S, Ni N. Topical treatment for capillary hemangioma of the eyelid using beta-blocker solution. Arch Ophthalmol. 2010 Feb;128(2):255-6. doi: 10.1001/archophthalmol.2009.370. No abstract available.
Other Identifiers
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AVL-SWS-TT4PWM
Identifier Type: -
Identifier Source: org_study_id
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