Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1 Associated Cutaneous Neurofibromas (cNFs)
试验速览
- 阶段
- 不适用
- 状态
- 暂停
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Treated cutaneous neurofibromas (cNFs)
Participants will receive treatment with laser at a wavelength of 755 nm to the cutaneous Neurofibromas lesion.
干预措施: GentleMax Pro (with skin cooling) (Device)
Treated cutaneous neurofibromas (cNFs)
Participants will receive treatment with laser at a wavelength of 755 nm to the cutaneous Neurofibromas lesion.
干预措施: GentleMax Pro (without skin cooling) (Device)
Control cutaneous neurofibromas (cNFs)
A complementary region of cNFs of similar characteristics to the treatment area in the same body region will be selected to serve as an untreated control group.
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
时间窗: Anytime from initiation of treatment through study completion, an average of 8 months.
Treatment will be considered tolerable if \<40% of participants treated have a \>Grade 2 CTCAE v5 adverse event (AE).
次要结局
- Patient Global Assessment of cNF Improvement (Likert Scale)(Baseline, Day 90, and 30 days after final treatment (up to Day 240).)
- Patient Satisfaction(Baseline, Day 90, and 30 days after final treatment (up to Day 240).)
- Clinician Global Assessment of cNF Improvement (Likert Scale)(Baseline, Day 90, and 30 days after final treatment (up to Day 240).)
- VAS Pain Scale(Baseline, every 30 days (up to Day 240).)
- Rate of Healing(Baseline, every 30 days (up to Day 240).)
- cNF Appearance (Height)(Baseline, Day 90, and 30 days after final treatment (up to Day 240).)
- cNF Appearance (Volume)(Baseline, Day 90, and 30 days after final treatment (up to Day 240).)
研究者
Richard Rox Anderson, MD
Director, Wellman Center for Photomedicine
Massachusetts General Hospital