E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10010331 |
E.1.2 | Term | Congenital, familial and genetic disorders |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 effect of VX-770 on hyperpolarized helium-3 magnetic resonance imaging (3He-MRI) in subjects aged 12 years and older with CF who have the G551D-CFTR mutation on at least 1 allele |
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E.2.2 | Secondary objectives of the trial |
- To evaluate the safety of VX-770 in subjects aged 12 years and older with CF who have the G551D-CFTR mutation on at least 1 allele - To evaluate the efficacy of VX-770 in subjects aged 12 years and older with CF who have the G551D-CFTR mutation on at least 1 allele |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male or female with Cystic Fibrosis - Must have the G551D-CFTR mutation on at least 1 allele - FEV1 ≥40% of predicted normal for age, gender, and height at Screening - 12 years of age or older - Must be able to swallow tablets |
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E.4 | Principal exclusion criteria |
- History of solid organ or hematological transplantation - Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening - Use of inhaled hypertonic saline treatment within 14 days prior to the Screening Visit - Extensive body tattoos or other physical features that will confound MRI |
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E.5 End points |
E.5.1 | Primary end point(s) |
Part A: Change from Day 15 Visit to Day 43 Visit in total ventilation defect defined by 3He-MRI Part B: Change from baseline through Week 48 in total ventilation defect defined by 3He-MRI |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Part A: Day 15 Visit to Day 43 Part B: Baseline through Week 48 |
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E.5.2 | Secondary end point(s) |
Part A: - Safety as determined by adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), and vital signs Efficacy as determined by: - Absolute change from Day 15 Visit to Day 43 Visit in percent predicted FEV1 - Change from Day 15 Visit to Day 43 Visit in sweat chloride - Change from Day 15 Visit to Day 43 Visit in Cystic Fibrosis Questionnaire-Revised (CFQ-R) score
Part B: - Safety as determined by adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital ECGs, and vital signs Efficacy as determined by: - Absolute change from baseline through Week 48 in percent predicted FEV1 - Change from baseline through Week 48 in sweat chloride - Change from baseline through Week 48 in CFQ-R score |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Part A: Safety: through Day 57 Other endpoints: change from Day 15 to day 43
Part B: Safety: through week 48 Other endpoints: baseline to wk 48 |
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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 | No |
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 | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
Will this trial be conducted at a single site globally?
| Yes |
E.8.4 | Will this trial be conducted at multiple sites globally? | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA 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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 2 |