An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 61
- 试验地点
- 17
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability to provide written informed consent (parental/guardian consent and participant assent if less than [<] 18 years of age).
- •Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
- •Body weight greater than or equal to (≥) 16 kilograms (kg).
- •Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
- •Confirmed laboratory values within the central laboratory ranges at screening.
- •In male and female participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period.
- •Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions.
排除标准
- •Chronic use of systemic tobramycin within 4 weeks prior to screening.
- •Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
- •Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening and randomization.
- •Known hypersensitivity to any of the ingredients or excipients of the study drug.
- •Exposure to another investigational drug within 4 weeks prior to screening.
- •Treatment with intravenous antibiotics within 3 weeks prior to screening.
- •History of solid organ or hematological transplantation.
- •Ongoing immunosuppressive therapy (other than corticosteroids).
- •Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.
- •Known portal hypertension.
- •Pregnancy or breast-feeding.
研究组 & 干预措施
Ataluren
Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.
干预措施: Ataluren (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
时间窗: Baseline (Day 1) up to end of study (Week 196)
AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Number of Participants With Clinically Significant Laboratory Abnormalities
时间窗: Baseline (Day 1) up to end of study (Week 196)
Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.
次要结局
- Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria(Baseline up to Week 192)
- Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria(Baseline up to Week 192)
- Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria(Baseline up to Week 192)
- Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry(Baseline, Week 192)
- Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)(Baseline, Week 196)
- Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG(Baseline, Week 196)
- Change From Baseline in Vital Signs at Final Visit (Week 196)(Baseline, Week 196)