Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: An Open-label Multicenter Extension Study
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 115
- 试验地点
- 26
- 主要终点
- Formation of New Ulcers
研究概览
简要总结
This open label extension trial will allow ongoing treatment of subjects who participated in the randomized controlled trials, and will provide long term information about the safety of treprostinil diethanolamine SR in subjects with SSc and digital ulcers.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Eligible subjects who completed assessments for the final visit of the previous TDE-DU-201 controlled trial and who signed appropriate informed consent were eligible to enroll in this extension study.
- •If a female of childbearing potential, the subject must have agreed to continue practicing an acceptable method of birth control (i.e., surgical sterilization, approved hormonal contraceptives, barrier methods [such as a condom or diaphragm] used with a spermicide, or an intrauterine device).
排除标准
- •Have had any change in clinical status that, in the opinion of the Investigator, would pose a safety risk for participation in this extension study;
- •Have been found to be unable to complete study assessments in the previous controlled trial;
- •Have prematurely discontinued study drug during the previous study due to clinical worsening or treatment related adverse events.
研究组 & 干预措施
Treprostinil
Treprostinil diethanolamine sustained release tablet initiated at 0.25 mg and titrated up to a maximum dose of 16 mg BID or the individual's maximum tolerated dose (MTD).
干预措施: treprostinil diethanolamine (Drug)
结局指标
主要结局
Formation of New Ulcers
时间窗: 18 months (or last study visit)
The number and percentage of subjects who developed new ulcers during the study were summarized.
Net Ulcer Burden- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment
时间窗: Baseline and Months 1, 3, 6, 9, 12, and 18
Net ulcer burden at any given assessment was defined as the number of "new" or "active" ulcers at that assessment, plus the number of "indeterminate" ulcers at that assessment that had previously been classified as either "active" or "new" at any earlier assessment during the study. The mean change in net ulcer burden from time of study entry was summarized for each scheduled visit assessment.
Total Ulcer Number- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment
时间窗: Baseline and Months 1, 3, 6, 9, 12, and 18
The total ulcer number includes all ulcers designated as "active", "indeterminate", or "new" for a given visit. The mean change in the total number of ulcers present from time of study entry was summarized for each scheduled visit assessment.
次要结局
- Patient Function and Quality of Life Measure: Cochin Hand Function Scale (CHFS)-Mean Change From Study Entry in CHFS Score at Each Scheduled Assessment(Baseline and Months 1, 3, 6, and 12)
- Patient Function and QOL Measure: Scleroderma Health Assessments Questionnaire (SHAQ)- Mean Change From Study Entry in SHAQ Component Scores at Each Scheduled Assessment(Baseline and Months 1, 3, 6, and 12)