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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT03090971
• Date of registration: 08/03/2017
• Prospective Registration: No
• Primary sponsor: Fundação Educacional Serra dos Órgãos
• Public title: Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1
• Scientific title: Clinical Assessment of the Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With Neurofibromatosis Type 1
• Date of first enrolment: February 15, 2017
• Target sample size: 7
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03090971
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Brazil

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cutaneous Neurofibroma

Neurofibromatosis 1

Intervention(s)

Drug: Saline Solution

Drug: Diclofenac Sodium

Primary Outcome(s)

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

Secondary Outcome(s)

Efficacy - neurofibroma detatchment [Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period]

Efficacy - neurofibroma size [Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period]

Efficacy - presence of tissue necrosis in treated neurofibromas [Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period]

Safety - Adverse events [Time Frame: Throughout the 7-day treatment period and subsequent 30-day follow-up period]

Secondary ID(s)

NX101-02-2016

Source(s) of Monetary Support

Please refer to primary and secondary sponsors