Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 1
- 主要终点
- Evaluation of the degree of changes in disfigurement measures
研究概览
简要总结
Background:
People with Neurofibromatosis type 1 (NF1) have an increased risk of developing plexiform neurofibromas (PNs). PNs are tumors that form in the tissue. They can form anywhere in the body. They can become visible and cause deformations. Researchers want to see if selumetinib changes how PNs look in people with NF1. They also want to test a rating system for the visibility of these tumors.
Objective:
To see if treatment with selumetinib can improve the appearance of visible PNs in people with NF1, as determined by people who are/are not familiar with NF1.
Eligibility:
People with NF1 who have one or more visible PNs and have been enrolled in study 11C0161 or 08C0079. Clinicians and non-clinicians with and without experience in NF1 are also needed to serve as raters.
Design:
Participants are people with NF1 who had photos taken on study 11C0161 or 08C0079. Raters are people who will evaluate the PNs in the photos. They will rate the tumors on a scale from 1 to 10, from less to most visible.
Participants medical records will be reviewed. Their photos will be shown to 28 raters.
Raters will fill out a survey about their demographics, place of work, and if they are familiar with NF1. They will view sample photos to learn how PNs look and how to rate PNs.
Raters will view photos of PNs taken before and after selumetinib treatment. They will also view photos of PNs that were not treated. They will rate PNs for up to 40 participants. They will have 1-2 sessions. Each session will last 1 hour....
详细描述
Background:
Up to 50% of subjects with Neurofibromatosis Type 1 (NF1) will develop histologically benign plexiform neurofibromas (PN).
One of the most common morbidities associated with PN is a disfigurement that has the potential to pose formidable obstacles to the medical and psychosocial well-being of subjects with NF1.
The phase 2 trial of the MEK inhibitor selumetinib for inoperable PN (NCT01362803) have shown volumetric shrinkage of PN in approximately 74% of subjects with at least one PN morbidity, with most of the responsive tumors decreasing in size by 20-50% volume from baseline. Both trials have completed enrollment and primary study objectives.
In this selumetinib trial, standardized photography of visible PN was performed over time to assess for changes in disfigurement over time. This trial did not include criteria to measure changes in disfigurement and no validated criteria for measurement of PN-related disfigurement exist.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Evaluation of the degree of changes in disfigurement measures
时间窗: ongoing
Mean scores for disfigurement as determined by raters reviewing photographs before treatment and just prior to cycle 13 of treatment.
次要结局
- Factors most important in causing change in disfigurement(ongoing)
- Differences in the disfigurement scores assigned by raters between cohorts of clinical raters who differ by NF1 familiarity and non-clinical raters who differ by NF1 familiarity(ongoing)
- Overall difference among the 4 cohorts of raters with respect to scores assigned for disfigurement(ongoing)
- Feasibility of understanding and completing a novel disfigurement scale(ongoing)
- Correlation between achieving a partial response on selumetinib and having an improvement in PN-related disfigurement(ongoing)
- Intra-reliability of a novel disfigurement scale among raters(ongoing)
- Correlation between the degree of change in disfigurement and percentage of PN volume shrinkage(ongoing)
- Agreement among raters within a cohort with respect to the evaluation of disfigurement to assess the reliability of a novel disfigurement scale(ongoing)
- PN-related disfigurement rating consistency with subject/parent responses to Global Impression of Change (GIC)(ongoing)