Cutaneous NeurofibromacNF-HFU2101NCT05119582
HIFU Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and Efficacy
A study testing TOOsonix System ONE-M in people with Cutaneous Neurofibroma, run by Joergen Serup at 2 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cutaneous Neurofibroma
- What is being tested?
- TOOsonix System ONE-M
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 13 Apr 2022to 17 Jan 2024
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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 known allergy to injectable anesthetics (relevant for biopsy only).
- Patients with any condition which, in the Investigator's opinion, would make it unsafe (for the participant or study personnel) to treat the participant as part of this research study.
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- Patients, where target treatment may cause the acoustic beam to enter the eye.
- Pregnant females, due to possible discomfort with the procedure even though the procedure is localized and there is no new drug.
- Patients with tendency for keloid and hypertrophic scar formation .
- Patients with impaired wound healing.
- Patients with any other acute or chronic condition which, in the opinion of the investigator, could interfere with the conduct of the study.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: High-Intensity Focused Ultrasound (HIFU), a Novel Method for Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and Efficacy
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.
Read more
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.
- Conditions
- Cutaneous Neurofibroma
- Being tested
- TOOsonix System ONE-M
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TOOsonix System ONE-MDevice
Selected neurofibromas will be treated by high intensity focused ultrasound. Untreated fibromas will be used for controls.
| Study group | Role | What they receive |
|---|---|---|
| TOOsonix System ONE-M | Experimental | Cutaneous neurofibromas will be treated by high intensity focused ultrasound. Receives: Device: TOOsonix System ONE-M |
What is being measured
The primary outcome is the main question the study is designed to answer.
Short Term Safety Profile - Adverse Events
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.
Measured: 3 months
Long Term Safety Profile - Healing
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.
Measured: 9 months
Secondary outcomes (1)
Long Term Safety Profile - Adverse Events
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.
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2021
- Study start13 Apr 2022
- Primary completion17 Jan 2024
- Study completion17 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cutaneous NeurofibromaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The study is a multicenter (two-center) open-label study with high-intensity focused ultrasound (HIFU) treatment of patients with the genetic disease Neurofibromatosis Type1. The study is not comparative or randomized. The investigation is performed prior to CE mark to confirm safety and performance of the System ONE-M device when used as intended, i.e. as an non-invasive method to remove cutaneous neurofibromas by 20 MHz high intensity focused ultrasound.
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Joergen Serup
- Sponsor type
- University, hospital or other
- Collaborators
- Sahlgrenska University Hospital
- Responsible party
- Sponsor-investigator, Joergen Serup, Bispebjerg Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05119582
- Sponsor's protocol ID
- CIV-21-09-037759
- Acronym
- cNF-HFU2101
- Status last verified
- Jul 2024
- First submitted
- 29 Oct 2021
- Last updated
- 16 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High-Intensity Focused Ultrasound (HIFU), a Novel Method for Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and Efficacy
- ClinicalTrials.gov ID
- NCT05119582
- Acronym / sponsor ID
- cNF-HFU2101 / CIV-21-09-037759
- Conditions
- Cutaneous Neurofibroma
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Joergen SerupUniversity, hospital or other
- Collaborators
- Sahlgrenska University Hospital
- Study start
- 13 Apr 2022 (actual)
- Primary completion
- 17 Jan 2024 (actual)
- Study completion
- 17 Jan 2024 (actual)
- First posted
- 15 Nov 2021 (actual)
- Results
- Posted 16 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The study is a multicenter (two-center) open-label study with high-intensity focused ultrasound (HIFU) treatment of patients with the genetic disease Neurofibromatosis Type1. The study is not comparative or randomized. The investigation is performed prior to CE mark to confirm safety and performance of the System ONE-M device when used as intended, i.e. as an non-invasive method to remove cutaneous neurofibromas by 20 MHz high intensity focused ultrasound.
| Arm | Type | Receives |
|---|---|---|
| TOOsonix System ONE-M | Experimental | Device: TOOsonix System ONE-MCutaneous neurofibromas will be treated by high intensity focused ultrasound. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (11 listed)
- 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 known allergy to injectable anesthetics (relevant for biopsy only).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TOOsonix System ONE-MSelected neurofibromas will be treated by high intensity focused ultrasound. Untreated fibromas will be used for controls. | Device | TOOsonix System ONE-M |
Primary outcome
Short Term Safety Profile - Adverse Events
Time frame: 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.
Long Term Safety Profile - Healing
Time frame: 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.
1 secondary outcome
Long Term Safety Profile - Adverse Events
Time frame: 9 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing TOOsonix System ONE-M in people with Cutaneous Neurofibroma, run by Joergen Serup at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Denmark, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Short Term Safety Profile - Adverse Events (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2024.