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NCT05119582: cNF-HFU2101

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

Completed NA Results posted Last updated 16 October 2024
What this trial tests

NA trial testing TOOsonix System ONE-M in Cutaneous Neurofibroma in 20 participants. Completed in 17 January 2024.

Timeline
13 April 2022
Primary endpoint
17 January 2024
17 January 2024

Quick facts

Lead sponsorJoergen Serup
PhaseNA
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment20
Start date13 April 2022
Primary completion17 January 2024
Estimated completion17 January 2024
Sites2 locations across Denmark, Sweden

Drugs / interventions tested

Conditions studied

Sponsor

Joergen Serup

Who can join

18 and older, any sex, with Cutaneous Neurofibroma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Short Term Safety Profile - Adverse Events Primary · 3 months

The grade of any adverse events (AE) requiring medical intervention within three months of treatment should be low. Device-based treatment will be considered tolerable if less than 30% of participants treated have an "Overall AE Score" larger than Grade 2 AE at the 3-month follow-up (visit 4). Measured by a grading scale based on Common Terminology Criteria for Adverse Events (CTCAE), U.S. Department of health and human services, Ver 5.0, Nov 27, 2017.

GroupValue95% CI
TOOsonix System ONE-M0
TOOsonix System ONE-M20
Long Term Safety Profile - Healing Primary · 9 months

The rate and nature of spontaneous healing of the treated cNF lesion and any safety related event including potential wound formation and wound healing should be equivalent to expectations from alternative method(s). Safety evaluation includes rating of treatment-associated sequele by end of study, e.g. dyspigmentation and scarring and overall investigator rating compared to expectations. Measured on 5-point grading from Very Unsatisfied to Very Satisfied.

Larger tumor size (<5)
GroupValue95% CI
TOOsonix System ONE-M6
Unchaged (5)
GroupValue95% CI
TOOsonix System ONE-M16
Some reduction (6-7)
GroupValue95% CI
TOOsonix System ONE-M25
Major reduction or no longer visible (8-10
GroupValue95% CI
TOOsonix System ONE-M45
Long Term Safety Profile - Adverse Events Secondary · 9 months

Any objective adverse effect or event, local or systemic, related to the treatment and the investigational device. Measured by a grading scale based on Common Terminology Criteria for Adverse Events (CTCAE), U.S. Department of health and human services, Ver 5.0, Nov 27, 2017.

Participants with an "Overall AE Score" larger than Grade 2 at the 9-month follow-up (visit 7)
GroupValue95% CI
TOOsonix System ONE-M0
Participants with an "Overall AE Score" of Grade 2 or below at the 9-month follow-up (visit 7).
GroupValue95% CI
TOOsonix System ONE-M20

Sponsor's own description

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.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Immunomodulation and targeted drug delivery with high intensity focused ultrasound (HIFU): Principles and mechanisms.
    Ashar H, Ranjan A. · · 2023 · cited 41× · PMID 36965581 · DOI 10.1016/j.pharmthera.2023.108393
  2. [Treatment and progress of cutaneous neurofibroma].
    Zhu B, Wei C, Wang W, Gu B, et al · · 2022 · cited 2× · PMID 36111466 · DOI 10.7507/1002-1892.202205072
  3. [Plastic surgical treatment of neurofibromatosis type 1].
    Längle G, Gohritz A, Gstöttner C, Harnoncourt L, et al · · 2025 · PMID 39875738 · DOI 10.1007/s00104-024-02232-5

Verify or expand the search:

Other trials of TOOsonix System ONE-M

Trials testing the same drug.

Other recruiting trials for Cutaneous Neurofibroma

Currently open trials in the same condition.

Other Joergen Serup trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05119582.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing