Trial Outcomes & Findings for HIFU Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: Safety and Efficacy (NCT NCT05119582)
NCT ID: NCT05119582
Last Updated: 2024-10-16
Results Overview
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.
COMPLETED
NA
20 participants
3 months
2024-10-16
Participant Flow
Participant milestones
| Measure |
TOOsonix System ONE-M
Cutaneous neurofibromas will be treated by high intensity focused ultrasound.
TOOsonix System ONE-M: Selected neurofibromas will be treated by high intensity focused ultrasound.
Untreated fibromas will be used for controls.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
TOOsonix System ONE-M
n=20 Participants
Cutaneous neurofibromas will be treated by high intensity focused ultrasound.
TOOsonix System ONE-M: Selected neurofibromas will be treated by high intensity focused ultrasound.
Untreated fibromas will be used for controls.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=20 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=20 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=20 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=20 Participants
|
|
Region of Enrollment
Sweden
|
10 participants
n=20 Participants
|
|
Region of Enrollment
Denmark
|
10 participants
n=20 Participants
|
PRIMARY outcome
Timeframe: 3 monthsPopulation: The grade of any adverse events (AE) requiring medical intervention within three months of treatment was measured.
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.
Outcome measures
| Measure |
TOOsonix System ONE-M
n=20 Participants
Cutaneous neurofibromas will be treated by high intensity focused ultrasound.
TOOsonix System ONE-M: Selected neurofibromas will be treated by high intensity focused ultrasound.
Untreated fibromas will be used for controls.
|
|---|---|
|
Short Term Safety Profile - Adverse Events
Participants with an "Overall AE Score" larger than Grade 2 at the 3-month follow-up (visit 4).
|
0 Participants
|
|
Short Term Safety Profile - Adverse Events
Participants with an "Overall AE Score" of Grade 2 or below at the 3-month follow-up (visit 4).
|
20 Participants
|
PRIMARY outcome
Timeframe: 9 monthsPopulation: Tumor size was clinically assesed relative to baseline at the 9-month follow-up visit (visit 7). The assessment was based on tumor size alone, e.g. investigators assessment of width, elevation, and volume. \<5: larger; 5: unchanged; 6-7: some reduction; 8-10: major reduction or no longer visible.
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.
Outcome measures
| Measure |
TOOsonix System ONE-M
n=92 Tumors
Cutaneous neurofibromas will be treated by high intensity focused ultrasound.
TOOsonix System ONE-M: Selected neurofibromas will be treated by high intensity focused ultrasound.
Untreated fibromas will be used for controls.
|
|---|---|
|
Long Term Safety Profile - Healing
Larger tumor size (<5)
|
6 Tumors
|
|
Long Term Safety Profile - Healing
Unchaged (5)
|
16 Tumors
|
|
Long Term Safety Profile - Healing
Some reduction (6-7)
|
25 Tumors
|
|
Long Term Safety Profile - Healing
Major reduction or no longer visible (8-10
|
45 Tumors
|
SECONDARY outcome
Timeframe: 9 monthsPopulation: The grade of any adverse events (AE) requiring medical intervention within nine months of treatment was measured.
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.
Outcome measures
| Measure |
TOOsonix System ONE-M
n=20 Participants
Cutaneous neurofibromas will be treated by high intensity focused ultrasound.
TOOsonix System ONE-M: Selected neurofibromas will be treated by high intensity focused ultrasound.
Untreated fibromas will be used for controls.
|
|---|---|
|
Long Term Safety Profile - Adverse Events
Participants with an "Overall AE Score" larger than Grade 2 at the 9-month follow-up (visit 7)
|
0 Participants
|
|
Long Term Safety Profile - Adverse Events
Participants with an "Overall AE Score" of Grade 2 or below at the 9-month follow-up (visit 7).
|
20 Participants
|
Adverse Events
TOOsonix System ONE-M
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place