An Open Label, Long-term, Safety, and Tolerability Extension Study Using the Pediatric Formulation of Bosentan in the Treatment of Children With Idiopathic or Familial Pulmonary Arterial Hypertension Who Completed FUTURE 1
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 33
- 主要终点
- Change From Baseline to End of Study (EOS) in Height for Age.
研究概览
简要总结
The main objective of the FUTURE 2 study was to assess the long-term safety and tolerability of the pediatric formulation of bosentan in children with idiopathic pulmonary arterial hypertension or familial pulmonary arterial hypertension who completed FUTURE 1 study.
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
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent by the parents or the legal representatives.
- •Patients who completed the FUTURE 1 study.
- •Patients who tolerated bosentan pediatric formulation and for whom bosentan is considered beneficial at the end of FUTURE
- •Males or females >= 2 and < 12 years of age at enrollment in FUTURE 2 (this study). Females who are menstruating must have a negative pregnancy test. A reliable method of contraception must be considered, if appropriate.
排除标准
- •Intolerance to bosentan despite dose reductions.
- •Any clinically significant laboratory abnormality that precludes continuation of bosentan therapy.
- •Pregnancy or breast-feeding.
- •Known hypersensitivity to bosentan or any of the excipients.
- •Premature and permanent study drug discontinuation during FUTURE 1.
研究组 & 干预措施
Bosentan
Bosentan was administered at 4 mg/kg twice daily (b.i.d.) until the end of the study. It could be down-titrated to 2 mg/kg b.i.d. if not well tolerated.
干预措施: Bosentan (Drug)
结局指标
主要结局
Change From Baseline to End of Study (EOS) in Height for Age.
时间窗: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
In order to compare the growth data with those of healthy children, growth curves are calculated from height data collected throughout the follow-up period. For each patient, height measured at each study visit was converted to a z-score and expressed in standard deviations (SD) from WHO growth standards. The Z-score was calculated according to the following formula: Z-score = (observed value of the study participant - median value of the reference population) / SD value of the reference population
Change From Baseline to End of Study (EOS) in Body Weight
时间窗: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including growth as measured by changes from baseline in body weight and height.
Change From Baseline to End of Study (EOS) in Systolic Blood Pressure (SBP)
时间窗: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in blood pressure.
Change From Baseline to End of Study (EOS) in Diastolic Blood Pressure (DBP)
时间窗: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in blood pressure.
Change From Baseline to End of Study (EOS) in Pulse Rate
时间窗: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in pulse rate.
Proportion of Patients With Treatment-emergent Liver Function Abnormalities
时间窗: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average
The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including laboratory abnormalities related to liver enzymes. Proportion of patients with increase in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) above 3 times upper limit of normal (ULN) is reported here.
Proportion of Patients With Treatment-emergent Hemoglobin Abnormalities
时间窗: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average
The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including hemoglobin abnormalities. Proportion of patients with marked hemoglobin decreases (i.e., decrease of or above 15% of the lower normal limit (LL)) is reported here.
Number of Subjects With Adverse Events Leading to Premature Discontinuation of Study Treatment
时间窗: From the first study drug administration in FUTURE 1, for an average of 31 months
次要结局
未报告次要终点