EUCTR2015-003291-77-DE进行中（未招募）1 期
A phase IIa, open label study of multiple doses of GLPG1837 in subjects with cystic fibrosis and the G551D mutation
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Galapagos NV
- 入组人数
- 32
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. Male or female subjects = 18 years of age, on the day of signing the Informed Consent Form (ICF), with a confirmed diagnosis of cystic fibrosis:
- •a.Clinical diagnosis of cystic fibrosis with signs/symptoms involving at least two organ systems, and
- •b.Medical history of elevated sweat chloride =60 mmol/L by quantitative pilocarpine iontophoresis (documented in the subject’s medical record) or 2 disease causing CFTR mutations (documented in the subject’s medical record).
- •2.Gating G551D CFTR mutation on at least one allele in the CFTR gene (documented in the subject’s medical record or CF registry); any known or unknown mutation allowed on the 2nd allele. Subject inclusion can be performed, provided that genotype information is available in source data.
- •3.Subject must meet one of the following:
- •a.Subjects currently receiving treatment with ivacaftor must be on a stable regimen for at least 2 weeks prior to screening;
- •b.Subjects not on a treatment regimen with ivacaftor for at least 2 weeks prior to screening.
- •4.Weight = 40.0 kg.
- •5.Subjects on stable concomitant treatment regimen for at least 4 weeks prior to baseline (excluding ivacaftor), as defined in the study protocol.
- •6.Pre- or post-bronchodilator FEV1 = 40% of predicted normal for age, gender and height at screening.
- •7.Female subjects must have a negative blood pregnancy test. Determination of serum follicle-stimulating hormone (FSH) will be done for any suspected postmenopausal female with at least 12 months of continuous spontaneous amenorrhea, with FSH levels > 40 IU/mL being confirmative for menopause. For hysterectomy and tubal ligation, documented confirmation will be requested.
- •8.Subjects will have to use highly effective contraceptive methods prior to the first dose of the study drug, during the study, and for at least 12 weeks after the last dose of the study drug.
- •a.If the subject is a sexually active woman of childbearing potential, she and her male partner are required to simultaneously use 2 effective contraceptive methods as defined in the protocol. Hormonal contraceptives will not be considered as an effective method; however, female subjects are not required to discontinue hormonal contraceptives. Female subjects who use contraception must have done so for at least 14 days prior to the first dose of the study drug.
- •b.Non-vasectomized sexually active male subjects with female partners of childbearing potential must be willing to use a condom in addition to having their female partner use another form of contraception as defined in the protocol.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 27
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1.History of sensitivity to any component of the study drug, or a history of drug or other allergy that, in the investigator’s opinion, contraindicates the subject’s participation in the study.
- •2.On an ivacaftor-containing treatment regimen and unable or unwilling to discontinue ivacaftor for the washout and treatment periods of the study.
- •3.A history of a clinically meaningful unstable or uncontrolled chronic disease including underlying cystic fibrosis that makes the subject unsuitable for inclusion in the study in the opinion of the investigator.
- •4.Liver cirrhosis and portal hypertension.
- •5.History of malignancy within the past 5 years (except for carcinoma in situ of the uterine cervix and basal cell carcinoma of the skin that has been treated with no evidence of recurrence).
- •6.Any significant change in the medical regimen (including dose or frequency) for pulmonary health within 4 weeks of baseline, including: antibiotics, corticosteroids, inhaled bronchodilators, hypertonic saline, mannitol or dornase alfa; ibuprofen and airway clearance techniques. Individuals taking inhaled antibiotics for suppression of chronic airways infection must be on a stable regimen for at least 8 weeks prior to baseline and willing to continue the same antibiotic through Day 29.
- •7.Unstable pulmonary status or respiratory tract infection (including pulmonary exacerbation), based on the investigator’s opinion, or changes in therapy for pulmonary disease within 4 weeks of baseline; as defined in the protocol.
- •8.History of lung volume reduction surgery or lung transplant.
- •9.Use of continuous (24 hours per day) supplemental oxygen therapy.
- •10.Clinically significant abnormalities detected on electrocardiogram (ECG) regarding either rhythm or conduction (e.g., QTcF = 450 ms, or a known long QT syndrome). A first degree heart block will not be considered as a significant abnormality.
- •11.Use of medication known to prolong the QT interval (including herbal and naturopathic therapy).
- •12.History of solid organ or haematological transplantation or currently on a transplantation waiting list.
- •13.Abnormal liver function defined as aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma glutamyl transferase (GGT) > 3 x upper limit of the normal range or bilirubin > 2x upper limit of the normal range.
- •14.Abnormal renal function defined as creatinine clearance < 50mL/min using the Cockroft-Gault equation.
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