ISRCTN96750019
Active, not recruiting
Phase 2
A phase IIb, multicentre, randomised, double-blind, placebo-controlled, crossover study to evaluate the efficacy and safety of dirocaftor/posenacaftor/nesolicaftor in subjects with cystic fibrosis aged 18 years or older
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Treatment of Cystic Fibrosis (CF) in patients with CFTR mutations who are responders in the organoid assay
- Sponsor
- niversity Medical Center Utrecht
- Enrollment
- 52
- Status
- Active, not recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Male or female subjects who completed the HIT\-CF Organoid Study and are 18 years of age or older on the date of informed consent
- •2\. Confirmed diagnosis of CF as follows:
- •2\.1\. Sweat chloride value of \=60 mmol/L based on quantitative pilocarpine iontophoresis (at screening) OR 2 CF\-causing mutations
- •2\.2\. chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
- •3\. Clinically stable CF disease in the opinion of the investigator with no significant changes in health status within 28 days prior to Day 1
- •4\. Forced expiratory volume in one second (FEV1\) \=40% of predicted to \=90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI) \-2012 multi\-ethnic all\-age reference equations
- •5\. Body mass index (BMI) \=16 kg/m² and \=30 kg/m²
- •6\. Non\-smoker and non\-tobacco user (including all inhalational nicotine delivery systems) for a minimum of 30 days prior to screening, and subject agrees not to smoke or use tobacco for the duration of the study
- •7\. Subjects of childbearing potential must meet contraception requirements
- •8\. Willing to remain on a stable medication regimen for CF from 28 days before Day 1 through the last study visit
Exclusion Criteria
- •1\. Subject has at least one of the following CFTR\-mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849\+10kbC\>T OR A combination of any two of the following mutations: any nonsense mutation, 1717\-1G\>A, 621\+1G\>T, 3120\+1G\>A, 1898\+1G\-\>A, CFTRdele2,3, and 2183AA\-\>G
- •2\. History or current evidence of any clinically significant cardiac (eg, heart failure, left ventricular hypertrophy, myocardial infarction, and arrhythmia), endocrinologic, hematologic, hepatobiliary (eg, clinically significant cirrhosis with or without portal hypertension), immunologic, metabolic, urologic, pulmonary (besides CF), neurologic (eg, subarachnoid haemorrhage, intracranial haemorrhage, cerebrovascular accident, intracranial trauma, and
- •autonomic neuropathy), dermatologic, psychiatric, renal, or other major disease, that is unstable or could interfere with the subject’s participation in or completion of the study, in the opinion of the investigator
- •3\. Clinically significant screening results that would exclude subject from the study (eg, medical history, physical examination, ECG, vital sign, pulse oximetry, and laboratory profiles) or any conditions that, would make the subject unsuitable for enrolment or could interfere with the subject’s participation in or completion of the study, in the opinion of the investigator. The medical monitor must be contacted for review of any subjects with screening results or conditions that may make them unsuitable for enrolment or could interfere with participation in or completion of the study.
- •4\. Prolonged QTcF \>450 msec at screening
- •5\. Abnormal liver function as defined by AST, ALT, gamma\-glutamyl transferase (GGT), or alkaline phosphatase \=3 times or total bilirubin \=2 times upper limit of the normal range
- •6\. Haemoglobin \<10 g/dL
- •7\. Platelet count \<150,000 cells/mm³
- •8\. Abnormal renal function at screening defined as creatinine clearance \<60 mL/min using the Modified Diet in Renal Disease (MDRD)
- •9\. Hospitalisation, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness (in the opinion of the investigator) requiring an increase or addition of medication, such as antibiotics or corticosteroids, within 28 days of Day 1
Outcomes
Primary Outcomes
Not specified
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