NL-OMON43806已完成2 期
A phase IIa, open-label study of two doses of GLPG1837 in subjects with cystic fibrosis and the S1251N mutation - Galapagos, Saphira 2
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Galapagos NV
- 入组人数
- 4
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
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
- •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 S1251N 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
- •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 2weeks 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).
- •6.Pre- or post-bronchodilator FEV1 >= 40% of predicted normal for age,
- •gender, 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 listed 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 listed
- •in the protocol.
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
- •3.Concomitant use of antifungal drugs (e.g. itraconazole, ketoconazole,
- •voriconazole, posaconazole) within 4 weeks of baseline.
- •4.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.
- •5.Liver cirrhosis and portal hypertension.
- •6.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).
- •7.Any significant change in the medical regimen (including dose and
- •frequency) for pulmonary health within 4 weeks of baseline, including:
- •antibiotics; corticosteroids (as defined in the protocol); 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.
- •8.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.
- •9.History of lung volume reduction surgery or lung transplant.
- •10.Use of continuous (24 hours per day) supplemental oxygen therapy.
- •11.Clinically significant abnormalities detected on electrocardiogram
- •(ECG) regarding either rhythm or conduction (e.g., QTcF >= 450 ms, or aknown long QT syndrome). A first degree heart block will not be
- •considered as a significant abnormality.
- •12.Use of medication known to prolong the QT interval (including herbal
- •and naturopathic therapy).
- •13.History of solid organ or haematological transplantation or currently
- •on a transplantation waiting list.
- •14.Abnormal liver function defined as aspartate aminotransferase (AST),
- •alanine aminotransferase (ALT), GGT > 3 x upper limit of the normal
- •range or bilirubin > 2 x upper limit of the normal range.
- •15.Abnormal renal function defined as creatinine clearance < 50mL/min
- •using the Cockroft-Gault equation.
研究者
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