E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic fibrosis (CF) Non-CF Bronchiectasis |
|
E.1.1.1 | Medical condition in easily understood language |
Cystic fibrosis and bronchiectasis are diseases that can both affect the lungs, leading to recurrent chest infections and lung scarring. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10006445 |
E.1.2 | Term | Bronchiectasis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
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 |
We plan to assess the value of novel MRI techniques to image the lungs of people with CF and bronchiectasis. Our main aims are to see how they compare to current measurements of lung function/structure and check that they give us useful information. Overall, we hope to identify the best MRI scanning methods for assessment of these lung diseases and employ those techniques in future clinical trials.
In CF, there is great interest in newer treatments that can improve lung function. At present, we use relatively simple measures such as blowing tests to assess and monitor the efficacy of these therapies. These measurements may not always be sensitive enough to detect changes in the lungs, so newer tests that are more sensitive are required. The lung disease in bronchiectasis is similar to CF and so studying both patient groups will highlight the utility of lung MRI in a broader population.
Following this study, we aim to run a clinical trial that will use our lung MRI scanning expertise |
|
E.2.2 | Secondary objectives of the trial |
The secondary objectives of this study are to assess how feasible the different types of MRI scans are for our participants and check how consistent the scans are between different study visits.
We will perform different types of MRI scans and what we learn about participant comfort/tolerability as well as imaging quality will greatly assist in the design of future lung MRI studies. We are not sure how much the different MRI scans will change when repeated over time. As such, we will invite participants to be scanned on multiple occasions and then compare their lung images longitudinally.
|
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Project title: An integrated omics approach to characterise the microbiome of the Cystic Fibrosis airways Anticipated start date - January 2020 Objective: To characterise the CF microbiome using 16S rRNA gene sequencing, metagenomics, metabolomics and metatranscriptomics.
|
|
E.3 | Principal inclusion criteria |
For this study, eligibility criteria have been separated into workstreams as follows: Workstream 1 – Adult Healthy Volunteers Workstream 2 – Adults with CF Workstream 3 – Adults with Bronchiectasis Workstream 4 – Children aged 12 to less than 16 years with CF
Workstream 1 Inclusion criteria: (Adult Healthy Volunteers) • Aged 16 years and over • For women, a negative urinary pregnancy test at screening and study visits • No significant respiratory disease or other significant medical condition within the last year
Workstreams 2 and 3 Inclusion criteria: (Adults with diagnosed Cystic Fibrosis or non-CF Bronchiectasis) • Aged 16 years and over • For women, a negative urinary pregnancy test at screening and study visits • Diagnosis of cystic fibrosis by a respiratory physician (workstream 2) or bronchiectasis by computer tomography (CT) scan or a respiratory physician (workstream 3) • Clinically stable as defined by respiratory care team
Workstream 4 Inclusion criteria: (Paediatric patients with diagnosed Cystic Fibrosis) • Parent or legal guardian able to give informed consent • Aged 12 years to less than 16 years • For females of child bearing age, a negative urinary pregnancy test at screening and study visits • Diagnosis of cystic fibrosis by a paediatric respiratory physician |
|
E.4 | Principal exclusion criteria |
Workstream 1 Exclusion criteria: (Adult Healthy Volunteers) • Standard MRI exclusion criteria – a history of implanted / indwelling magnetically active material • Acute respiratory illness within 30 days of MRI • Subject has received an Investigational Medical Product (not including hyperpolarized 129Xe) within 30 days of MRI and administration of 129Xe deemed inappropriate in context of other study • Subject not deemed fit enough to tolerate procedure • Subject deemed unsuitable by clinical investigator for other reasons
Workstreams 2 and 3 Exclusion criteria: (Adults with diagnosed Cystic Fibrosis or non-CF Bronchiectasis) • Standard MRI exclusion criteria – a history of implanted / indwelling magnetically active material • A current pulmonary exacerbation considered to be of sufficient severity to prevent safe MRI scanning • Current infection or previous infection within the last 12 months with Burkholderia cepacia complex or Mycobacterium abscessus • Previous lung transplantation • Evidence of susceptibility to or established hypercapnic (type 2) respiratory failure • Subject has received an IMP (not including hyperpolarized 129Xe) within 30 days of MRI and administration of 129Xe deemed inappropriate in context of other study • Subject not deemed fit enough to tolerate procedure • Subject deemed unsuitable by clinical investigator for other reasons
Workstream 4 Exclusion criteria: (Paediatric patients with diagnosed Cystic Fibrosis) • Standard MRI exclusion criteria – a history of implanted / indwelling magnetically active material • A current pulmonary exacerbation considered to be of sufficient severity to prevent safe MRI scanning • Current or previous infection/colonization with Burkholderia cepacia complex or Mycobacteria abscessus • Previous lung transplantation • Evidence of susceptibility to or established hypercapnic (type 2) respiratory failure • Subject has received an IMP (not including hyperpolarized 129Xe) within 30 days of MRI and administration of 129Xe deemed inappropriate in context of other study • Subject not deemed fit enough to tolerate procedure • Subject deemed unsuitable by clinical investigator for other reasons |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Success of acquiring 129Xe, OE and UTE MRI lung images from patients with CF and non-CF Bronchiectasis and produce baseline data for these groups to inform future clinical studies. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
We aim to have completed recruitment and imaging of all volunteers within 36 months of the start date of the study. The end of the study will be the last visit of the last participant. Other analysis will continue with a programme determined by the technology and software available and research findings. |
|
E.5.2 | Secondary end point(s) |
1) To assess the ability of participants of each group to adhere to the protocols, thus informing the study design of future trials
2) To assess repeatability of MR outcomes across study visits for those subjects undertaking more than one MR scanning visit.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
The same timepoints for both primary and secondary outcomes will be used (see above). |
|
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 | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 0 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 0 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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
|
The trial will end immediately after the last visit of the last subject undergoing the trial. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 1 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 1 |