Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Time to disease progression
研究概览
简要总结
The investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type 1 (NF1).
The investigators also wish to measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
详细描述
Neurofibromas contain a large quantity of fibrous matter, and it is not anticipated that significant reductions in tumor size can be achieved in large, long established, tumors. Cutaneous neurofibromas, which do not usually become apparent until puberty, continue to increase in size and number throughout adulthood. The psychosocial burden of these disfiguring tumors is significant, and the targeted age group (14-30) is at a life period associated with an acceleration in tumor growth. It is for these reasons that the investigators hope to affect the growth rate of less established tumors, in order to prevent or lessen this burden as the patient progresses further into adulthood.
Therefore, the investigators wish to determine the time to disease progression (defined as 50% growth in size over baseline) for benign neurofibromas treated with PDT in patients with neurofibromatosis type 1 (NF1) in subjects aged 14-30.
The treatment will consist of choosing several neurofibromas of similar size, and applying a topical drug called Levulan, or just the topical application alone (a placebo). Within 24 hours of drug treatment, the neurofibromas will be illuminated with red light (both Levulan and placebo). The Levulan is what is known as a photosensitizer, and will be activated by the red light to potentially kill some of the tumor cells. Approximately every 6 months after, for three years, the tumors will be measured by digital photography and ultrasound to see if they are growing more slowly than the ones with the placebo application alone.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient is 14 years or older.
- •Diagnosed NF1, determined by American Academy of Neurology Guidelines (see Diagnosis section).
- •Tumor Location: cutaneous, trunk, or limbs only.
- •Tumor Type: superficial dermal neurofibromas ≤4mm deep.
- •Patient has provided written informed consent.
- •Patient is willing to and can comply with study follow-up requirements.
- •Absence of any other malignancy.
排除标准
- •Life expectancy less than 3 years.
- •Cutaneous photosensitivity to the wavelengths used to active PDT.
- •A diagnosis of porphyria.
- •Allergy to aminolevulinic acid or any of the topical solution vehicle components.
- •Previous chemotherapy within 6 weeks of proposed PDT.
- •Other concurrent tumor therapy.
研究组 & 干预措施
PDT Treatment
Each subject will have either placebo or Levulan Kerastick topical application applied to matched sets of neurofibromas. Each subject will have both sets, in order to serve as his/her own control subject.
16 to 24 hours post study drug treatment, both sets of neurofibromas, Levulan and placebo treated, will be irradiated with red light (630 nm) from an Omnilux Revive light device at 100 mW/cm2 for 1000 seconds (16.7 minutes).
干预措施: aminolevulinic acid (Drug)
结局指标
主要结局
Time to disease progression
时间窗: 3 years
The time it takes for 50% growth in tumor size over baseline measurements.
次要结局
- Tumor growth rate(3 years)
研究者
Donald Basel
Professor
Medical College of Wisconsin