E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011764 |
E.1.2 | Term | Cystic fibrosis NOS |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of VX-809 in CF patients who are homozygous for the DeltaF508-CFTR mutation |
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E.2.2 | Secondary objectives of the trial |
To evaluate the effect of VX-809 on biomarkers of CFTR activity, pulmonary function, and patient-reported outcomes (PROs) in subjects with CF who are homozygous for the DeltaF508-CFTR mutation
To evaluate the pharmacokinetics of VX-809 in subjects with CF who are homozygous for the DeltaF508-CFTR mutation
To determine a dose of VX-809 for further clinical study
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The most important inclusion criteria are: (1) Male or female with confirmed diagnosis of CF; must be accompanied by a sweat chloride value greater than or equal to 60 mmol/L or a sweat sodium greater than or equal to 60 mmol/L, by quantitative pilocarpine iontophoresis, on at least 1 occasion. (2) Must have the DeltaF508-CFTR mutation in both alleles. (4) Age 18 years or older. (5) Weight greater than or equal to 40 kg. (6) Body mass index (BMI) greater than or equal to 18.5. (8) FEV1 greater than or equal to 40% of predicted normal for age, gender, and height (Knudson standards). (9) Oxygen saturation (pulse oximetry) greater than or equal to 92% on room air. (13) Willing to remain on a stable medication regimen for the duration of study participation. |
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E.4 | Principal exclusion criteria |
The most important exclusion criteria are: (1) History of any illness that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject. (2) Ongoing acute illness including acute respiratory or lower respiratory infections. Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug. (3) Pregnant, planning a pregnancy, or breast-feeding. (6) Liver function test result greater than or equal to 3x upper limit of normal (ULN). (7) History of abnormal renal function: in the past year creatinine clearance less than 50 mL/min using Cockcroft-Gault equation. (8) History of prolongation of QT/QTcF interval (greater than 450 msec in males, greater than 470 msec in females). (10) History of solid organ or hematological transplantation. (12) Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout. (13) Illness within 14 days before receiving the first dose of study drug. “Illness” is defined as a recent non-serious, non-acute condition, e.g., common cold. (14) Treatment with systemic antibiotics for an acute illness within 14 days before receiving the first dose of study drug. (15) Concomitant use of any inhibitors or inducers of CYP3A4. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability assessments based on adverse events, hematology, clinical chemistry, urinalysis, electrocardiograms (ECGs), vital signs, and physical examinations. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the study will occur when the data base is locked and the data from the study are unblinded. Data base lock is estimated to occur approximately 2 and 1/2 months after the last patient completes the study. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 10 |