Clinical trial NCT06934928: Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs)
Official title: Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1 Associated Cutaneous Neurofibromas (cNFs)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Neurofibromatosis 1 (NF1)
Neurofibromatosis Type I
Keywords: Neurofibromatosis
NF1
Cutaneous Neurofibroma
cNF
GentleMax Pro
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Johns Hopkins University
First posted: 2025-04-18
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of Alexandrite (755 nm) Laser in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are:

Will performing up to 6 months of treatment sessions with alexandrite laser will result in tolerable local skin reactions and reduction in both individual cNF size by \>50% as well as improved cNF appearance in the treated field? If there is a comparison group: Researchers will compare laser treatment with cooling to both laser treatment without cooling and an untreated control see if laser treatments are effective and if cooling makes treatment more tolerable.

Participants will:

* Receive up to 6 monthly laser treatments.
* Complete surveys asking about pain during and after treatments.
* Complete surveys asking about satisfaction with the treatments.
* Undergo 2D photography and 3D imaging of treatment areas.
* Optionally, receive biopsies of up to 6 treated lesions to investigate characteristics of tumors that respond well to treatment as well as non-respondent tumors.


Eligibility criteria: Inclusion Criteria:

* Participant is an adult ≥18 years of age.
* Participant has a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
* Family history of NF1
* Six or more light brown ("cafe-au-lait") spots on the skin
* Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas
* Freckling under the arms or in the groin area
* Two or more pigmented, benign bumps on the eye's iris (Lisch nodules)
* A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dysplasia of long bones, often in the lower leg
* Tumor on the optic nerve that may interfere with vision
* Participant is seeking treatment for cNF.
* Participants must have ≥ 12 paired cNF (6 to be treated without DCD, 6 to be treated with DCD) that are visible and measure at least 2 mm in size in the target treatment area. The target treatment area must be amenable to both laser treatments and surveillance with digital and 3D photography. Preferred locations are trunk (back or chest), arms and legs.
* Participant is able and willing to comply with all visit, treatment, and evaluation schedules and requirements.
* Participant is able to understand and provide written informed consent.
* Participant has no concurrent injury or wound in the target area.

Exclusion Criteria:

* Participant cannot give informed consent or adhere to study schedule.
* Participant is Fitzpatrick skin type V-VI.
* Participant is actively tanning during the course of the study.
* For female participants: participant is pregnant.
* Participant has 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.

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
timeFrame: Anytime between initiation of treatment and study completion.
Secondary outcomes:
measure: Patient Global Assessment of cNF Improvement (Likert Scale)
timeFrame: Baseline, Day 90, and 30 days after final treatment (up to Day 240).
measure: Clinician Global Assessment of cNF Improvement (Likert Scale)
timeFrame: Baseline, Day 90, and 30 days after final treatment (up to Day 240).
measure: VAS Pain Scale
timeFrame: Baseline, every 30 days (up to Day 240).
measure: Patient Satisfaction
timeFrame: Baseline, Day 90, and 30 days after final treatment (up to Day 240).
measure: Rate of Healing
timeFrame: Baseline, every 30 days (up to Day 240).
measure: cNF Appearance (Height)
timeFrame: Baseline, Day 90, and 30 days after final treatment (up to Day 240).
measure: cNF Appearance (Volume)
timeFrame: Baseline, Day 90, and 30 days after final treatment (up to Day 240).
Other outcomes:
measure: Biologic Effect/Histology
timeFrame: 3 months after final treatment session.
Locations:
city: Boston
country: United States
facility: Wellman Center for Photomedicine, Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Richard R. Anderson, MD
role: PRINCIPAL_INVESTIGATOR
References: