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Cystic Fibrosis (CF) clinical trials

View clinical trials related to Cystic Fibrosis (CF).

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NCT ID: NCT05818319 Recruiting - Clinical trials for Cystic Fibrosis (CF)

Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Urine After a Short Pause of Therapy

Start date: June 1, 2023
Phase: N/A
Study type: Interventional

In cystic fibrosis (CF) renal base excretion is impaired, due to mutations in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene, since CFTR function is crucial in regulation of the kidney's HCO3- excretion. The investigators suggest that challenged urine HCO3- excretion is a biomarker of CFTR function, which can be used to evaluate the extent of CFTR dysfunction and the possible correcting effects of CFTR modulating therapy. This study aims to evaluate changes in challenged urine HCO3- excretion in CF patients, who are currently in treatment with the triple CFTR modulator combination therapy, Elexacaftor/tezacaftor/ivacaftor (ETI), before, during, and after a short treatment pause.

NCT ID: NCT04853368 Terminated - Clinical trials for Cystic Fibrosis (CF)

Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis

Start date: September 20, 2021
Phase: Phase 2
Study type: Interventional

Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40. The main objective of this study is to assess how safe and effective is the combination therapy of galicaftor/navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 in adult participants with CF who are homozygous or heterozygous for the F508del mutation in each arm. Galicaftor/Navocaftor/ABBV-119 combination therapy and Galicaftor/Navocaftor/ABBV-576 is being developed as an investigational drug for the treatment of CF. Study doctors place participants in 1 of the 4 groups, called treatment arms. Each group receives a different treatment. Around 90 adult participants with a diagnosis of CF will be enrolled in the study around approximately 35 sites worldwide. Participants in arm 1 will receive oral capsules of galicaftor/navocaftor dual combination for 28 days followed by galicaftor/navocaftor/ABBV-119 triple combination for 28 days. Participants in arms 2 and 3 will receive the galicaftor/navocaftor/ABBV-119 triple combination or placebo for 28 days. Participants in arm 4 will receive galicaftor/navocaftor/ABBV-576 triple combination therapy for 28 days. For all study arms, ABBV-576, galicaftor, navocaftor, will be given once daily and ABBV-119 twice a day. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

NCT ID: NCT04375878 Completed - Clinical trials for Cystic Fibrosis (CF)

OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic Fibrosis

Start date: July 20, 2020
Phase: Phase 2
Study type: Interventional

The primary objectives of this study are to assess the safety and efficacy of MS1819 in enteric capsules vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). The exploratory objective of the extension phase (EP) is to find a dose of MS1819 in immediate release capsules that is safe and results in CFA values in a therapeutic range.

NCT ID: NCT03746483 Completed - Clinical trials for Cystic Fibrosis (CF)

OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

OPTION
Start date: January 10, 2019
Phase: Phase 2
Study type: Interventional

The primary objectives of this study are to assess the safety and efficacy of MS1819-SD vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).

NCT ID: NCT03597347 Terminated - Clinical trials for Mycobacterium Infections, Nontuberculous

Trial of Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial Infection

ENCORE
Start date: June 20, 2019
Phase: Phase 2
Study type: Interventional

A study to evaluate the efficacy of inhaled molgramostim administered open-label to adult cystic fibrosis (CF) subjects with chronic pulmonary nontuberculous mycobacterial (NTM) infection, with or without ongoing antimycobacterial guideline based combination therapy.

NCT ID: NCT03445793 Completed - Clinical trials for Cystic Fibrosis (CF)

: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)

Start date: March 1, 2018
Phase:
Study type: Observational

This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva. Clinical and laboratory measurements will be measured at baseline, 1 month, 3 months, and 6 months after initiation of tez/iva. Change from baseline at 6 months pre-specified will be reported. The length of study participation will be approximately 6 months.

NCT ID: NCT03234387 Terminated - Clinical trials for Cystic Fibrosis-related Diabetes

A CFit Study - Baseline

Start date: November 17, 2017
Phase:
Study type: Observational

A great medical success is the increase in the median survival age associated with cystic fibrosis (CF). However, this success has led to a new era of research with the aim to maximise the quality of life (QoL) of the aging CF population. Recent research has demonstrated that the traditional method of determining disease progression, i.e. pulmonary function, no longer adequately predict survival rates. Therefore, various bodies have promoted cardiopulmonary exercise testing (CPET), as outcomes from this test (e.g. one's maximal O2 uptake [VO₂max]) are known predictors of the QoL, risk of hospitalisation and prognosis of individuals with CF. One of the most common non-pulmonary co-morbidities of CF is CF-related diabetes (CFRD). Importantly, CFRD is associated with a poorer pulmonary function compared to CF patients without CFRD, and ultimately a worsened prognosis. Despite this, the influence an impaired glycaemic control has upon the VO₂max derived from a CPET is unknown in CF. Therefore, the present study aims to assess whether VO₂max, an established determinant of QoL, differs between patients with CF with and without established CFRD as well as a group of age- and gender-matched healthy control subjects. The additional measures within the present study, such as: biomarkers of inflammation, redox balance and nitric oxide (NO2) bioavailability, as well as functional measures of microvascular endothelial function will aid our knowledge of the physiological abnormalities which are a cause or consequence of CFRD. Importantly, by identifying the factors which may contribute to CFRD progression and those that are viable for early intervention, mean the aims and objectives of this study are compatible with the top 10 research objectives set by the CF Trust.

NCT ID: NCT02778750 Completed - Clinical trials for Cystic Fibrosis (CF)

Evaluation Of The Pan-microbiome and Host Immune Response in CF

Start date: June 5, 2015
Phase:
Study type: Observational

Investigators will examine temporal and regional dynamic changes in the microbiome of Cystic Fibrosis patients to explore microbiome features that are associated with an inflammatory phenotype. Investigators hypothesize that temporal and spatial differences in lung microbiome are associated with host inflammatory responses. While chronic and polymicrobial airway colonization are commonly recognized in cystic fibrosis (CF), it is unclear what factors of the microbial environment lead to infection with pathogenic microorganism. This is a multi center, longitudinal cohort of adult Cystic Fibrosis subjects recruit4ed from NYU and Columbia to understand how changes in the airway microbiome may affect the host inflammatory responses in Cystic Fibrosis (CF). There will be three approaches to understanding inflammatory responses; 1) a longitudinal assessment of temporal changes in the microbiome over a 6-month period of clinical stability; 2) comparison of the regional differences in airway microbiome between lung segments with more versus less disease; 3) evaluation of functional aspects of the lung microbiome.

NCT ID: NCT02605590 Completed - Clinical trials for Cystic Fibrosis (CF)

Safety, Tolerability and Pharmacokinetics Study of AIR DNAse Administered by Inhalation to Healthy Adult Volunteers

Start date: December 2015
Phase: Phase 1
Study type: Interventional

This study will be performed to assess the safety, tolerability and PK of escalating inhaled AIR-DNase doses administered as a single dose followed by multiple doses for 5 consecutive days in healthy subjects. A thorough review of safety data will be conducted after completion of each dose level per subject and prior to both, moving to the next dose level in Part 1 and commencement of the multiple dose Part 2.

NCT ID: NCT02163681 Completed - Healthy Clinical Trials

MRI for Non-Invasive Imaging in Neonates and Children

Start date: January 1, 2011
Phase: N/A
Study type: Interventional

The purpose of this study is to develop rapid MRI techniques for imaging the lung with hyperpolarized helium-3 gas as an inhaled contrast agent. These techniques will be piloted in adults and older children before testing them in younger children and infants. The purpose is to enable imaging of non-sedated infants by imaging so fast as to freeze motion.