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Study details
Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Changes in this gene can affect the amount of protein made from this gene, or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky and cause problems in the lungs and other organs. This study will look at how well the medicine ‘VX-121’ works, in combination with other medicines. Early studies have suggested that taking these medicines may improve...
- Trials Tracker ID
- TT001996
- Age range
- 18 years and older
- Trial status
- Closed with results
- Therapeutic approach
- Restore CFTR Function
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Study details
ALX-009 is composed of hypothiocyanite ion (OSCN-) and lactoferrin, two substances that are naturally occurring components of the immune system in the lungs but are lacking in CF patients’ lungs. It has been demonstrated in vitro that OSCN- and lactoferrin are capable of killing all tested Gram(-) bacteria, including bad bugs resistant to available antibiotics. This clinical trial is split into four parts. People with CF or bronchiectasis are planned to be enrolled in the fourth part, the main purpose of which is to assess the safety of ALX-009 after repeated administration of increasing doses. The doses will be chosen...
Study updates
This trial was terminated.
Study results summary
This trial was terminated.
- Trials Tracker ID
- TT001986
- Age range
- 18 years and older
- Trial status
- Study discontinued early
- Therapeutic approach
- Anti-Infective
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Study details
Phase 1/2 safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of VX-121
- Trials Tracker ID
- TT001981
- Age range
- 18 years and older
- Trial status
- Closed with results
- Therapeutic approach
- Restore CFTR Function
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Study details
Adults with cystic fibrosis often have low iron levels (iron deficiency). This is partly due to poor absorption of iron from the gut, and partly to the trapping of iron within cells of the immune system during periods of infection. Unfortunately, iron tablets are often ineffective in this setting, and may cause significant side effects in patients with cystic fibrosis. In other patient groups, intravenous iron is used routinely to correct iron deficiency. In these patients it has been shown to be safe, and to improve energy levels, exercise tolerance, cognitive function and quality of life, even in the absence...
- Trials Tracker ID
- TT001976
- Age range
- 18 years and older
- Trial status
- Closed with results
- Therapeutic approach
- Nutritional-GI
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This trial has been archived
Study details
TRIAL RESULTS LINK: https://erj.ersjournals.com/content/56/suppl_64/1275 There are many oscillatory PEP devices on the market to use for chest physiotherapy, with strong evidence that they are effective treatments in clearing the lungs. However, they don’t provide the user feedback to confirm that they are using the correct technique or indicate when each blow or treatment session has been completed. As a result this can lead parents to have a lack of confidence in whether their child is completing the physiotherapy session correctly or completely.A father of a CF patient has developed a device alongside the CF team at Cambridge University Hospital called...
- Trials Tracker ID
- TT001971
- Age range
- Between 8 and 16 years
- Trial status
- Closed with results
- Therapeutic approach
- Other
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Study details
Airway clearance treatments (ACTs) and physical activity can mitigate the progression of CF lung disease, but these routine physiotherapy treatments are burdensome and adherence is low. Traditional research methods have failed to produce credible evidence to guide practice, partly because ‘blinding’ isn’t possible and patient preference can confound results. We have worked with engineers and designers to develop an electronically chipped sensor (Fizzyo sensor) which mounts onto standard widely used airway clearance devices. This sensor monitors breathing during routine airway clearance. Additionally wearable activity trackers can monitor physical activity. We can now facilitate automatic transmission of ACT and physical activity...
- Trials Tracker ID
- TT001966
- Age range
- Between 6 and 16 years
- Length of participation
- 14 months
- Trial status
- Closed with results
- Therapeutic approach
- Behavioural