EUCTR2013-004581-34-DE进行中（未招募）1 期
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 208
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
- •2. Age =6 years.
- •3. Body weight =16 kg.
- •4. Sweat chloride >60 mEq/L.
- •5. Documentation of the presence of a nonsense mutation in at least 1 allele of the CFTR gene, as determined by genotyping performed at a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization.
- •6. Verification that a blood sample has been drawn for sequencing of the CFTR gene.
- •7. Ability to perform a valid, reproducible spirometry test using the study-specific spirometer with demonstration of an FEV1 =40% and =90% of predicted for age, gender, and height.
- •8. Demonstration at Visit 2 of a valid %-predicted FEV1 within 15% of the Screening %-predicted FEV1 value.
- •9. Resting oxygen saturation (as measured by pulse oximetry) =92% on room air.
- •10. Confirmed screening laboratory values within the central laboratory ranges specified in Table 2 of the protocol.
- •11. In subjects 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.
- •12. Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 80
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 130
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Known hypersensitivity to any of the ingredients or excipients of the study drug (polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate).
- •2. Previous participation in the Phase 3 trial of ataluren (PTC124-GD-009-CF).
- •3. 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 or any change in acute therapy between screening and randomization.
- •4. Chronic use of inhaled or systemic tobramycin within 4 weeks to
- •5. Exposure to another investigational drug within 4 weeks prior to screening.
- •6. Ongoing participation in any other therapeutic clinical trial.
- •7. 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.
- •8. Treatment with intravenous antibiotics within 3 weeks prior to screening.
- •9. Ongoing immunosuppressive therapy (other than corticosteroids).
- •10. Ongoing warfarin, phenytoin, or tolbutamide therapy.
- •11. History of solid organ or hematological transplantation.
- •12. Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening.
- •13. Known portal hypertension.
- •14. Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test.
- •15. Pregnancy or breast-feeding.
- •16. Current smoker or a smoking history of =10 pack-years (number of cigarette packs/day × number of years smoked).
- •17. Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
研究者
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