CompletedNot Applicable

HIFU Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and Efficacy

Denmark, Sweden20 participantsStarted 2022-04-13

Plain-language summary

The overall objective is to demonstrate safety and efficacy of HIFU treatment of cutaneous neurofibromas located close to the surface of the skin in patients with the genetic disease Neurofibromatosis Type 1. The study will use a new investigational equipment that has been specially developed for dermatological therapy. The study includes 20 patients in total distributed between the two centers, each having a minimum of 8 cutaneous neurofibromas eligible for treatment. All participants are adults (over 18 years) of both sexes. The new treatment method is based on focusing intensive ultrasound just below the skin surface. This creates a very fast localized heating in small and very well-defined volumes containing neurofibroma tissue. This heating destroys or weakens the tissue, and the body's natural processes will subsequently transport affected cells away through the lymphatic and vascular systems. During the healing-process, the rejected tissue is replaced by new skin cells that are not expected to be fibrous. The treatment is intended to be carried out without breaking the skin surface, and open wounds are therefore avoided. This is an essential advantage of the method compared to all existing therapies, which are based on physical removal of tumors through an open skin surface (e.g. surgery or laser therapy). Complications with risk of pain, infection and scarring will therefore be significantly reduced with the new proposed method. The treatment is carried out by sending focused ultrasound from the handpiece of the equipment into the target area with neurofibromas. The equipment is set to send doses of approximately 150 milliseconds (0.15 seconds). The skin area and HIFU doses can be followed on the system computer screen and will be placed side-by-side with approximately 1-2 millimeter spacing. To achieve good energy transfer from handpiece to skin, ordinary ultrasound gel is used. There are no other special pre-treatments or preparations for the process. HIFU treatment is expected to be less painful than other treatments used. The treatment is quick, and typically takes less than 1 minute for a each area the size of a typical neurofibroma.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Males and females 18 years of age or older. * Patients with a clinical diagnosis of NF1 based on germline genetic testing or by meeting the internationally recognized criteria given by the Clinical Care Advisory Board of the National Neurofibromatosis Foundation (now the Children's Tumor Foundation) * Patients must be seeking active treatment for cNF. * Patients must have ≥ 8 paired cNF that are visible and measure a minimum of 2 mm in size. These must be in areas amenable to treatment and surveillance. * Patients with Fitzpatrick Type I to VI skin-type * Able and willing to comply with all visits, treatments, evaluations, schedules, and requirements. * Patients shall have received oral and written study information, accepted participation and signed the informed consent document. * Patients who are willing and mentally and physically capable to understand and follow the treatment and follow-up schedule including post-treatment care instructions. * Patients who are willing to have photographs and images taken of the treated lesions to be used anonymously or coded in evaluations and publications. Exclusion Criteria: * Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions. * Individuals who cannot give informed consent or adhere to study schedule. * Patients who are actively tanning during the course of the study. * Patients with adverse reactions to compounds of any external agent in use. * Patients with…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Short Term Safety Profile - Adverse Events

Timeframe: 3 months

Long Term Safety Profile - Healing

Timeframe: 9 months

Trial details

NCT IDNCT05119582

SponsorJoergen Serup

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-01-17

Results submitted2024-07-18

View on ClinicalTrials.gov More Cutaneous Neurofibroma trials