NCT06120036已完成1 期
Dosing and Tolerability of Deoxycholic Acid Versus Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 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
This study will evaluate the tolerability and effectiveness of two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera injection. Each patient will have a treatment and a control site.
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult males and females ≥18 years of age
- •Have 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
- •Patients must be seeking treatment for cNF
- •Patients must have ≥ 6 paired cNF per modality (3 treated and 3 untreated). cNF should be visible and measure between 2-8mm in size. These must be in areas amenable to treatment and surveillance with digital photography.
- •cNF must be located on the trunk, arms or legs of the patient
- •Able and willing to comply with all visit, treatment and evaluation schedules and requirements
- •Able to understand and provide written informed consent
排除标准
- •Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
- •Individuals who cannot give informed consent or adhere to study schedule
- •Actively tanning during the course of the study
- •Adverse reactions to compounds of any external agent (e.g., gels, lotions or anesthetic creams) required for use in the study, if no alternative to the said agent exists;
- •Known allergy to injectable anesthetics, polidocanol or deoxycholic acid
- •Those with acute thromboembolic diseases
- •Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy
- •Those with dysphagia
- •Women who are pregnant
- •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
Asclera Injection
Active Comparator
干预措施: Asclera (Drug)
Kybella Injection
Active Comparator
干预措施: Kybella (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: 3 months after treatment
Device based treatment will be considered tolerable if \<40% of participants treated have a \>grade 2 adverse event (AE). A grade 2 AE is defined as an adverse event that requires treatment.
次要结局
- Treatment specific patient reported outcomes (PRO)(Baseline, 1 day after treatment, 1 week after treatment, 1 month after treatment, 2 months after treatment, 3 months after treatment, 6 months after treatment, 12 months after treatment)
- Clinician reported outcomes (ClinRO)(Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment)
- Modified SkinDex for cNF(Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment)
研究者
Richard Rox Anderson, MD
Director, Wellman Center for Photomedicine
Massachusetts General Hospital
研究点 (1)
Loading locations...
相似试验
已完成
1 期
Phase 1-2 Study of Deoxycholic Acid Injection (ATX-101) for the Reduction of Submental FatModerate or Severe Submental FullnessNCT00618722Kythera Biopharmaceuticals85
已完成
2 期
Deoxycholic Acid Injection Submental Fat (SMF) Magnetic Resonance Imaging (MRI) and Subject-reported Outcome Measures StudyModerate or Severe Submental FullnessNCT01032889Kythera Biopharmaceuticals129
终止
3 期
Ursodeoxycholic Acid Plus Budesonide Versus Ursodeoxycholic Acid Alone in Primary Biliary Cirrhosis (PBC)Primary Biliary CirrhosisNCT00746486Dr. Falk Pharma GmbH62
已完成
1 期
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy VolunteersHealthy VolunteersNCT03263507Ionis Pharmaceuticals, Inc.32
已完成
2 期
Phase 2 Study of Deoxycholic Acid Injection (ATX-101) for the Reduction of Submental FatModerate or Severe Submental FullnessNCT00618618Kythera Biopharmaceuticals73