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Acute changes in respiratory signs and symptoms termed Pulmonary Exacerbations require treatment with intravenous antibiotics and hospital admission. These episodes cause substantial disruption to people's lives and impact on lung function, quality of life and lifespan. Current treatment regimes require improvement but further study is needed to identify who might benefit from a different approach. This observational study aims to assess if multi-dimensional measurements taken during treatment correspond with later treatment response. This may allow us to personalise treatment more effectively in the future and to better understand how individuals respond to treatment. As yet there is no model for...

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Trials Tracker ID
TT001641
Age range
18 years and older
Trial status
Recruitment complete
Therapeutic approach
Other
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A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation (VX15-770-124)

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This trial has been archived

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation

Ivacaftor was generally safe and well tolerated in children aged 4 to 24 months. Treatment for up to 24 weeks was associated with reductions in sweat chloride and improvements in the function of the pancreas. All adverse events reported were considered mild or moderate and likely not related to Ivacaftor treatment. 

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Trials Tracker ID
TT001611
Age range
Between 0 and 24 months
Trial status
Closed with results
Therapeutic approach
Restore CFTR Function
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Observational study of outcomes in cystic fibrosis patients with selected gating mutations

The study is being done to learn more about the effectiveness of Kalydeco in CF patients with the following specific gene mutaions: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. The study also looks to explore the effect of Kalydeco on your quality of life. This is an observational study. This means that your cystic fibrosis treatment and medical care will not change because you are participating in this study and the sponsor will not pay for Kalydeco. Your doctor will continue to make all decisions regarding your proper treatment and care. No part of this study is experimental.

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Trials Tracker ID
TT001606
Age range
6 years and older
Length of participation
48 months
Trial status
Recruitment complete
Therapeutic approach
Other
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Study of CTX 4430 in Cystic Fibrosis Patients

The purpose of this study is to assess the safety and tolerability of multiple oral doses of CTX-4430 when administered to cystic fibrosis (CF) patients once-daily (QD) for 15 days. This study will include two dose levels. For each dose level, blood samples will be collected for exploratory PK and PD assay validation. In addition, sputum will be collected for exploratory biomarker analysis. Following multiple dose administration, pulmonary function and exploratory lung clearance index (LCI) measurements will be taken.

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Trials Tracker ID
TT001601
Age range
Between 18 and 30 years
Length of participation
55 weeks
Trial status
Closed with results
Therapeutic approach
Anti-Inflammatory
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A study looking at the long term treatment of a CFTR modulator (VX-661) when taken with Ivacaftor

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This is a Phase 3, OpenLabel, Rollover study to look at how safe and effective longterm treatment with VX-661 together with Ivacaftor is in paticipants aged 12 years and older with cystic fibrosis (CF) that have a F508de-lCFTR mutation. The study involves treating all participants who meet all the rules of the study from one of the parent studies (VX-661-106,108,109 or 111) with a combination of VX-661 and Ivacaftor for approximately 96 weeks with a follow-up 28 days after the last dose. Approximately 1375 participants are potentially eligible to be enrolled. There is also an observational cohort (group) that will...

This study assessed the long-term safety and effectiveness of a combined CFTR modulator treatment TEZ/IVA in people with CF ages 12 and older who had either one or two copies of the F508del CFTR gene variant. The results of this study support the long-term use of TEZ/IVA treatment for people aged 12 years and older with CF who have either one or two copies of the F508del CFTR gene variant.

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Trials Tracker ID
TT001591
Age range
12 years and older
Length of participation
96 weeks
Trial status
Closed with results
Therapeutic approach
Restore CFTR Function

Cystic fibrosis physiotherapy

Physiotherapy is a crucial part of the daily treatment regime required to keep people with cystic fibrosis (CF) fit and healthy. Find out more.

Strategic Research Centre: How CF exocrine pancreatic disease may lead to CF related diabetes

Recent research suggests that cystic fibrosis-related diabetes (CFRD) is caused by damage to the digestive-juice-producing part of the pancreas, triggering things to go wrong in the insulin-producing ‘beta’ cells in another part of the pancreas. Researchers will investigate how the damage to insulin-producing cells occurs and the best ways to measure it in people with cystic fibrosis.

MHRA issues Drug Safety Update for Kaftrio

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The UK medicines regulator, the Medicines and Healthcare products Regulatory Agency (MHRA), have today published an update on Kaftrio highlighting a small increase in the risk of psychological side effects.