NCT01533376

Brief Summary

Primary Objective: • To assess the possible utility of topical timolol in the management of port-wine mark (PWM) in Sturge-Weber syndrome in children.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
3

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2012

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2012

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

February 11, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 15, 2012

Completed
6.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2019

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2019

Completed
Last Updated

March 21, 2019

Status Verified

March 1, 2019

Enrollment Period

6.9 years

First QC Date

February 11, 2012

Last Update Submit

March 19, 2019

Conditions

Sturge Weber SyndromePort-wine Mark

Keywords

Sturge WeberSWSTimololPort wine mark

Outcome Measures

Primary Outcomes (1)

  • Appearance of Port-wine Mark at treatment site

    Changes of color and size of PWM at treatment site will determine efficacy of the topical timolol.

    12 months

Study Arms (2)

Timolol

EXPERIMENTAL

Participants in this group will receive topical timolol

Drug: Timolol

Placebo

PLACEBO COMPARATOR

Participants in this group will receive Preservative free artificial tear gel.

Drug: Preservative free artificial tear gel.

Interventions

TimololDRUG

0.5% timolol maleate ophthalmic gel-forming solution applied once

Also known as: Timoptic-XE
Timolol
Preservative free artificial tear gel.DRUG

Preservative free artificial tear gel applied topically twice a day.

Placebo

Eligibility Criteria

Age2 Years - 10 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Wills Eye Institute

Philadelphia, Pennsylvania, 19107, United States

Location

Related Publications (15)

  • 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.

    PMID: 18349634BACKGROUND

  • 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.

    PMID: 20166161BACKGROUND

  • 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.

    PMID: 16722347BACKGROUND

  • 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.

    PMID: 20509816BACKGROUND

  • 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.

    PMID: 20013138BACKGROUND

  • 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.

    PMID: 20219318BACKGROUND

  • 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.

    PMID: 19296147BACKGROUND

  • 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.

    PMID: 16425790BACKGROUND

  • 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.

    PMID: 20085450BACKGROUND

  • 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.

    PMID: 18418641BACKGROUND

  • 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.

    PMID: 19588535BACKGROUND

  • 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.

    PMID: 19588532BACKGROUND

  • 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.

    PMID: 18550886BACKGROUND

  • 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.

    PMID: 19109584BACKGROUND

  • 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.

    PMID: 20142555BACKGROUND

MeSH Terms

Conditions

Sturge-Weber Syndrome

Interventions

Timolol

Condition Hierarchy (Ancestors)

HemangiomaNeoplasms, Vascular TissueNeoplasms by Histologic TypeNeoplasmsNeurocutaneous SyndromesNervous System DiseasesAngiomatosisVascular DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

PropanolaminesAmino AlcoholsAlcoholsOrganic ChemicalsPropanolsAminesThiadiazolesThiazolesSulfur CompoundsAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsMorpholinesOxazines

Study Officials

  • Alex V Levin, MD, MHSc

    Wills Eye Institute

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
CARE PROVIDER
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Chief, Pediatric Ophthalmology and Ocular Genetics

Study Record Dates

First Submitted

February 11, 2012

First Posted

February 15, 2012

Study Start

February 1, 2012

Primary Completion

January 1, 2019

Study Completion

February 1, 2019

Last Updated

March 21, 2019

Record last verified: 2019-03

Data Sharing

IPD Sharing
Will not share

Locations

US(1)