NCT06300502Enrolling By Invitation1 期
Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- Enrolling By Invitation
- 入组人数
- 15
- 试验地点
- 2
- 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 an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are:
Will performing:
- Up to 6 months treatment sessions
- A minimum of 30 days apart
- With up to 50 injections of deoxycholate into a maximum of 50 cNFs in a single region of the body (for a maximum total dose of 10 mL per monthly treatment session) 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?
Researchers will compare treated tumors and control tumors to see if the treatment is effective.
Participants will:
- Receive up to 6 monthly treatments with Kybella (deoxycholate). Treatment for a given tumor will be stopped when the tumor is assessed as clear clinically.
- 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
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（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.
- •Participant has ≥ 6 paired cNF that are visible and measure 2 mm or more in size. The target treatment area must be amenable to both deoxycholate injections 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 known allergy to deoxycholate.
- •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 actively tanning during the course of the study.
- •Participant has a known allergy to deoxycholic acid.
- •For female participants: those who are 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)
Active Comparator
Injection into up to 50 cutaneous Neurofibromas (for at total maximum of 10 mL injected) with Kybella (1% deoxycholic acid).
干预措施: Kybella (Drug)
Control cutaneous neurofibromas (cNFs)
No Intervention
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: 3 months after treatment
Treatment will be considered tolerable if \<40% of participants treated have a \>grade 2 CTCAE v5 adverse event (AE).
次要结局
- Patient Reported Outcomes(Baseline, after treatment session #4, and at 3 months after final treatment.)
- Clinician Reported Outcomes(Baseline, after treatment session #4, and at 3 months after final treatment.)
- VAS Pain Scale(Baseline, after each treatment session, 1 week after each treatment session, and 3 months after final treatment.)
- Patient Satisfaction(Baseline, after treatment session #4, and at 3 months after final treatment.)
- Rate of Healing(Baseline, 1 month, 2 months, 3 months, 4 months, 5 months, 6 months after baseline; 3 months after final treatment.)
- cNF Appearance (Height)(Baseline, after treatment session #4, and at 3 months after final treatment.)
- cNF Appearance (Volume)(Baseline, after treatment session #4, and at 3 months after final treatment.)
研究者
Richard Rox Anderson, MD
Principal Investigator
Massachusetts General Hospital
研究点 (2)
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