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

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Brief Title

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

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 Description

      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 place 7 days after the end of the treatment period and Assessment 6 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. Prior to any study-related procedure, written informed consent
      will be obtained from the participant. The Clinical Research From will be filled, stored,
      coded, and the data will be analyzed using GraphPad Prism, v. 5.0. Frequency tables will be
      generated and central tendencies calculated (mean, median, mode).
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Efficacy - presence of inflammatory process in the treated neurofibromas

Secondary Outcome

 Efficacy - presence of tissue necrosis in treated neurofibromas

Condition

Neurofibromatosis 1

Intervention

Diclofenac Sodium

Study Arms / Comparison Groups

 Cutaneous neurofibromas
Description:  Each subject will have two treatment neurofibromas and two control neurofibromas. Following microporation, the two treatment neurofibromas will be treated with topical diclofenac while the two control neurofibromas will be treated with topical saline.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

7

Start Date

February 15, 2017

Completion Date

June 30, 2017

Primary Completion Date

March 23, 2017

Eligibility Criteria

        Inclusion Criteria:

          -  Adults of both genders, between the ages of 18 and 65;

          -  NF1, diagnosed clinically by a neurologist, dermatologist, or other specialist
             knowledgeable about the disease, and defined as:

        A known mutation in the gene coding for neurofibromin

        or, the presence of 2 of the following 7 clinical manifestations of NF1:

          -  ≥ 6 café-au-lait macules on the body with diameters greater than 15mm in the greatest
             diameter;

          -  two or more neurofibromas of any type or one plexiform neurofibroma

          -  inguinal or axillary freckling

          -  two or more Lisch nodules (iris hamartomas)

          -  optic glioma

          -  a distinct osseous lesion, such as sphenoid wing dysplasia, pseudoarthrosis of the
             tibia, macrocephaly, or scoliosis

          -  a first-degree relative with NF1

          -  Presence of 4 or more cutaneous neurofibromas measuring 0.5-1.2cm in greatest
             diameter, present on thorax/abdomen or upper or lower limbs;

          -  If a woman of childbearing potential, is willing to use a medically acceptable form of
             contraception (in the judgment of the investigator) for the duration of the study;

          -  Is able to understand the informed consent form describing the risks of this study,
             and voluntarily signs the informed consent document;

          -  Is able to understand and comply with the requirements of the protocol.

        Exclusion Criteria:

          -  Surgical, medical, or investigative treatment for any of the 6 target cutaneous
             neurofibromas to be evaluated in the study within three months prior to the baseline
             visit;

          -  Active infection (bacterial, viral, or fungal) requiring systemic antibiotics within
             two weeks of the baseline visit;

          -  Pregnancy or breastfeeding;

          -  Immunocompromised because of a medical condition;

          -  Known hypersensitivity to diclofenac or any other NSAID;

          -  Known hypersensitivity to aspirin;

          -  has a known hypersensitivity to mannitol, sodium metabisulphite, benzyl alcohol, or
             propylene glycol;

          -  Known hypersensitivity to lidocaine;

          -  Currently receiving or has received with 2 weeks of screening an NSAID (including
             diclofenac), a COX-2 inhibitor, cyclosporine, methotrexate, an oral anti-diabetic,
             lithium, digoxin, diuretics, anticoagulants (such as warfarin), or a quinolone
             antibiotic; except for intralesional diclofenac, these medications will not be allowed
             during the study; low-dose aspirin used for cardioprotective effects will be allowed;

          -  Any history of hepatic (including hepatic porphyria) or renal disease resulting in
             ongoing compromised hepatic or renal function;

          -  History of a bleeding/coagulation disorder;

          -  History of gastrointestinal (gastric or intestinal) ulcer disease, Crohn's disease, or
             ulcerative colitis;

          -  Laboratory examination at screening that reveals in the opinion of the investigator
             significant, unstable, and/or untreated renal, hepatic, or metabolic
             disease/dysfunction;

          -  White blood cell count at screening that is less than 3000, or a platelet count at
             screening that is less than 150,000;

          -  Laboratory evaluation at screening that shows the hemoglobin lower than the lower
             limit of normal for the laboratory utilized;

          -  Under treatment for a medical condition that, in the opinion of the investigator, may
             interfere with the safety of the experimental treatment or with the evaluation of
             efficacy, including but not limited to cardiovascular and/or respiratory disease;

          -  Subject is not, in the opinion of the investigator, capable of giving informed consent
             to participate in the study;

          -  Subject has received an investigational therapy or procedure for any reason within 30
             days prior to screening.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Brazil

Location Countries

Brazil

Administrative Informations


NCT ID

NCT03090971

Organization ID

NX101-02-2016


Responsible Party

Principal Investigator

Study Sponsor

Fundação Educacional Serra dos Órgãos


Study Sponsor

, , 


Verification Date

October 2017