Long Term, Single-arm, Open-label Extension Study of Protocol AC-055-305 to Assess the Safety, Tolerability and Efficacy of Macitentan in Subjects With Eisenmenger Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 217
- 试验地点
- 51
- 主要终点
- Change in Borg Dyspnea Score at Month 6 and 12
研究概览
简要总结
Long-term study to evaluate if macitentan is safe, tolerable and efficient enough to be used for treatment of Eisenmenger syndrome.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with ES (including those with Down Syndrome) having completed the double-blind AC-055-305 / MAESTRO study as scheduled, i.e., who remained in the double-blind study up to Week 16 (whether or not they were still taking study drug at the end of this period).
排除标准
- •Subjects who prematurely discontinue double-blind study drug during the AC-055-305 / MAESTRO study due to:
- •an AE assessed as related to the use of study drug,
- •or elevated liver tests (related or unrelated to study drug).
研究组 & 干预措施
Macitentan
Macitentan 10 mg tablet, once daily.
干预措施: Macitentan 10 mg tablet, once daily. (Drug)
结局指标
主要结局
Change in Borg Dyspnea Score at Month 6 and 12
时间窗: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.
The Borg dyspnea score rates the severity of dyspnea (difficult or labored breathing) on a scale from 0 ('Nothing at all') to 10 ('Very, very severe - maximal'). For missing Borg dyspnea index values in the OL study, the following imputation rules were applied: If the reason for missing data was death, a value of 10 was imputed for all Borg visits from the date of death. For any other reasons, the last available value was carried forward.
Change in WHO Functional Class (FC) at Month 6 and 12
时间窗: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.
Class I: no symptoms with exercise or at rest. No limitation of activity. Class II: No symptoms at rest but slight limitation with ordinary activities causing symptoms (e.g. short of breath with climbing stairs). Class III: may not have symptoms at rest but activities greatly limited by shortness of breath, fatigue, or near fainting. Class IV: symptoms at rest and inability to carry out any physical activity without symptoms. Patients in class IV manifest signs of right heart failure. For missing WHO FC values in the OL study, the following imputation rules were applied: If the reason for missing data was death, class IV was imputed for all WHO visits from the date of death. For any other reasons, the last available value was carried forward.
Change in Exercise Capacity as Measured by 6-minute Walking Distance (6MWD) Month 6 and 12
时间窗: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.
NOTE: The MAESTRO-OL study was exploratory in nature and no primary efficacy and safety endpoint were defined in the clinical protocol. This and the other exploratory efficacy outcome measures posted were selected to be reported as a primary endpoints. All efficacy analyses were considered exploratory. The analyses of the exploratory efficacy endpoints focused on the absolute values and on the change from DB baseline to Week 16 in the DB study and to Month 6 and Month 12 in the OL study. For missing 6MWD values in the OL study, the following imputation rules were applied: If the reason for missing data was death, a distance of zero (0) meters was imputed for all 6MWD visits from the date of death. For any other reasons, the last available value was carried forward.
Change in Peripheral Oxygen Saturation (SpO2) at Rest at Month 6 and 12
时间窗: From baseline in DB parent study (AC-055-305, NCT01743001) up to month 12 in this OL study.
No imputation of missing data for SpO2 was applied. Oxygen saturation assessed by pulse oximetry: peripheral oxygen saturation (SpO2) at rest before the 6-minute walk test (6MWT)
次要结局
未报告次要终点