EUCTR2015-004917-26-NL进行中（未招募）1 期
A Phase 2a, 2-part, Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation With and Without Oral Ivacaftor in Subjects With Primary Ciliary Dyskinesia - PS-G202
Parion Sciences, Incorporated0 个研究点目标入组 123 人开始时间: 2016年7月28日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 123
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
入选标准
- •Subjects who meet all of the following inclusion criteria will be eligible.
- •1. Subject (or subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, where appropriate, assent form.
- •2. Willing and able to comply with scheduled visits, treatment plan, study restrictions laboratory tests, contraceptive guidelines, and other study procedures.
- •3. Willing and able to use the nebulization device as directed by the instructions for use.
- •4. The subject must have evidence supportive of a PCD diagnosis based on the following:
- •A. Subjects =12 to <18 years of age must meet 2 or more of the following PCD clinical criteria:
- •? Unexplained neonatal respiratory distress (at term birth) with need for respiratory support with CPAP and/or oxygen for >24 hours
- •? Any organ laterality defect confirmed by historical chest imaging – situs inversustotalis, situs ambiguous, or heterotaxy
- •? Daily, year-round wet or productive cough starting in first year of life or bronchiectasis on historical chest imaging
- •? Daily, year-round nasal congestion starting in first year of life or pansinusitis on historical sinus imaging
- •B. Subjects =18 years of age must have bronchiectasis on historical chest imaging
- •C. All subjects must ALSO have documentation of at least one of the following historical tests:
- •? For patients with no laterality defect, nNO level, measured with a chemiluminescent NO analyzer, during plateau <77 nL/min on 2 occasions, at least 2 months apart, with CF excluded by sweat chloride or genetic testing
- •? For patients with a laterality defect, nNO level, measured with a chemiluminescent NO analyzer, during plateau <77 nL/min on at least 1 occasion
- •? Diagnostic ciliary ultrastructural defect on transmission electron micrograph (TEM)
- •? 2 loss of function and/or known mutations in a single PCD-associated gene.
- •Prior to randomization, all subjects must have a confirmed diagnosis of PCD (including central review, as required) based on one of the following:
- •? 2 loss of function and/or known mutations in a single PCD-associated gene identified by the central genetic testing laboratory from the specimen obtained at the screening visit; previous genotype results cannot be used to determine eligibility for randomization.
- •? Diagnostic ciliary ultrastructural defect on transmission electron micrograph. A previously prepared TEM specimen will be reviewed centrally; a new specimen will not be obtained.
- •? Laterality defect that includes dextrocardia plus bronchiectasis in more than 1 lobe on historical chest imaging.
- •5. Subjects with percent predicted FEV1 of =40 to <90 percentage points adjusted for age, sex, and height according to the Global Lung Function Initiative (GLI) predicted values at the Screening Visit, taken 4 hours or more after last dose of short-acting bronchodilators (ß-agonists and/or
- •anticholinergics)
- •6. Non-smoker for the past 90 days prior to the Screening Visit and less than a 5 pack-year lifetime history of smoking, and willing to not smoke while enrolled in the study.
- •7. Stable regimen of medic
排除标准
- •Subjects who meet any of the following exclusion criteria will not be
- •1. Diagnosis of CF, including at least 1 of the following:
- •a. Documented sweat chloride test =60 mM by quantitative pilocarpine
- •iontophoresis
- •b. Abnormal nasal potential difference (NPD) test
- •c. 2 CF-causing mutations in the CFTR gene
- •2. Subjects with only 1 mutation in the CFTR gene and a sweat chloride
- •test =60 mM by quantitative pilocarpine iontophoresis.
- •3. History of any organ transplantation or lung resection or chest wall
- •4. Significant congenital heart defects, other than a laterality defect, at
- •the discretion of the investigator.
- •5. Diagnosis of Cri du chat syndrome (chromosome 5p deletion
- •6. Inability to withhold short-acting bronchodilator use for 4 hours prior
- •to clinic visit and long-acting bronchodilator use the night before the
- •first and last clinic visit of each treatment period.
- •7. History of any illness or any clinical condition that, in the opinion of
- •the investigator, might confound the results of the study or pose an
- •additional risk in administering study drug(s) to the subject. This may
- •include, but is not limited to history of clinically significant and
- •uncontrolled adrenal, neurologic, gastrointestinal, renal, hepatic,
- •cardiovascular (including hyper/hypotension and tachy/bradycardia),
- •psychological, pulmonary (other than PCD), metabolic, endocrine, or
- •hematological/coagulation disorder or disease, or scoliosis of such
- •severity that it impacts pulmonary function or any other major disorder
- •or disease, in the opinion of the investigator.
- •8. Use of diuretics (including amiloride) or renin-angiotensin
- •antihypertensive drugs (e.g., spironolactone, angiotensin converting
- •enzyme [ACE] and/or neural endopeptidase (NEP)-inhibitors, or
- •angiotensin receptor blockers [ARBs]) or trimethoprim or drospirenone
- •in the 28 days before Day 1 or anticipate need for these medications
- •during the study.
- •9. Had symptoms of acute upper or lower respiratory tract infection or
- •had an acute pulmonary exacerbation requiring treatment or was treated
- •with systemic antibiotics for ear or sinus disease within 28 days before
- •Day 1 (topical otic antibiotics allowed).
- •10. History of significant intolerance to inhaled HS, as determined by the
- •investigator.
- •11. History of drug or alcohol abuse, in the opinion of the investigator.
- •12. Known hypersensitivity to any of the study drugs or amiloride.
- •13. Used ivacaftor within 28 days prior to Day 1 or anticipate need for
- •ivacaftor during the study.
- •14. Pregnant and/or nursing females.
- •15. Any clinically significant laboratory abnormalities at the Screening
- •Visit as judged by the investigator, or any of the following:
研究者
相似试验
进行中（未招募）
1 期
CLEAN-PCD: Clearing Lungs with ENaC Inhibition in Primary Ciliary DyskinesiaPrimary Ciliary DyskinesiaMedDRA version: 20.0Level: PTClassification code 10069713Term: Primary ciliary dyskinesiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2015-004917-26-ITPARION SCIENCES, INC.123
进行中（未招募）
1 期
A study to evaluate whether VX-371 is safe and makes breathing easier in patients with primary ciliary dyskinesia (study also known as CLEAN-PCD or PS-G202)MedDRA version: 20.0 Level: PT Classification code 10069713 Term: Primary ciliary dyskinesia System Organ Class: 10010331 - Congenital, familial and genetic disordersPrimary Ciliary DyskinesiaEUCTR2015-004917-26-PLParion Sciences, Incorporated123
进行中（未招募）
1 期
A study to evaluate whether VX-371 is safe and makes breathing easier in patients with primary ciliary dyskinesia (study also known as CLEAN-PCD or PS-G202)MedDRA version: 20.0Level: PTClassification code 10069713Term: Primary ciliary dyskinesiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersPrimary Ciliary DyskinesiaEUCTR2015-004917-26-DKParion Sciences, Incorporated123
进行中（未招募）
1 期
A study to evaluate whether VX-371 is safe and makes breathing easier in patients with primary ciliary dyskinesia (study also known as CLEAN-PCD or PS-G202)Primary Ciliary DyskinesiaMedDRA version: 20.0Level: PTClassification code 10069713Term: Primary ciliary dyskinesiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2015-004917-26-DEParion Sciences, Incorporated123
已完成
2 期
A Phase 2a, Two-part, Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation With and Without Oral Ivacaftor in Subjects With Primary Ciliary DyskinesiaPrimary ciliary dyskinesiagenetic condition of the cilia10038686NL-OMON46905Parion Sciences, Incorporated15