Clinical Trial: Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Clinical Assessment of the Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With Neurofibromatosis Type 1

Brief Summary: This is an open, controlled, prospective, proof-of-concept study, in 7 patients presenting NF1 and cutaneous neurofibromas. This study will include three treatment visits to the study center and three follow-up visits. Treatment will consist of two stages: neurofibroma microporation using the laser device, followed by topical application of one drop of diclofenac 25mg/ml on the surface of the neurofibroma; followed by re-application of one drop of diclofenac, twice daily, for three days. The applications subsequent to the first application will be performed by the patients. Subjects will return to the study center at three day intervals (Assessments 2 & 3) for new microporation and topical diclofenac application, followed by at-home topical diclofenac application for three more days. Assessment 4 will take place 3 days after Assessment 3. Assessment 5 will take place 7 days after the end of the treatment period and Assessment 6 at 30 days after the last application of study drug. The primary efficacy variable in this study is the inflammatory process with the presence of tissue necrosis. The primary safety variable is the occurrence of adverse events considered to be associated with the study drug, occurring during the treatment period.

Detailed Summary: Neurofibromatosis type 1 (NF1) is an autosomal dominant neurocutaneous syndrome with highly variable clinical manifestations and that has a worldwide incidence of approximately 1/2500. The most common lesion is the cutaneous neurofibroma, appearing on the skin of 90% of adults with NF1. The number of cutaneous neurofibromas in an affected individual can vary from a few to several thousand. These lesions may be surgically removed, but typically recur, and surgical removal often leads to scarring. Intralesional administration of diclofenac was previously reported with favorable results, and significant inflammatory processes were observed within the treated neurofibromas, with tissue necrosis and detachment of some treated neurofibromas, effects that were not observed among the control neurofibromas. The primary objective of this study is to evaluate the use of topical diclofenac in the treatment of cutaneous neurofibromas in patients with NF1. The secondary objective of this study is to assess the safety of the use of topical diclofenac in the treatment of cutaneous neurofibromas in patients with NF1. This is an open, controlled, prospective, proof-of-concept study, in 7 patients presenting NF1 and cutaneous neurofibromas. This study will include three treatment visits to the study center and three follow-up visits. Treatment will consist of two stages: neurofibroma microporation using the laser device, followed by topical application of one drop of diclofenac 25mg/ml on the surface of the neurofibroma; followed by re-application of one drop of diclofenac, twice daily, for three days. The applications subsequent to the first application will be performed by the patients. Subjects will return to the study center at three day intervals (Assessments 2 & 3) for new microporation and topical diclofenac application, followed by at-home topical diclofenac application for three more days. Assessment 4 will take place 3 days after Assessment 3. Assessment 5 will take plac
Sponsor: Fundação Educacional Serra dos Órgãos

Current Primary Outcome: Efficacy - presence of inflammatory process in the treated neurofibromas [ Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period ]

Inflammatory process (redness, exculceration)


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Efficacy - presence of tissue necrosis in treated neurofibromas [ Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period ]
    Presence of tissue necrosis in treated neurofibromas
  • Efficacy - neurofibroma size [ Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period ]
    Reduction in neurofibroma size
  • Efficacy - neurofibroma detatchment [ Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period ]
    Detachment of the treated neurofibroma
  • Safety - Adverse events [ Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period ]
    Occurrence of adverse events considered to be associated with the study drug


Original Secondary Outcome: Same as current

Information By: Fundação Educacional Serra dos Órgãos

Dates:
Date Received: March 8, 2017
Date Started: February 15, 2017
Date Completion: June 30, 2017
Last Updated: March 20, 2017
Last Verified: March 2017