Patient screening in the ongoing phase 2 SPAN clinical trial researching a new enteric microgranulation formulation of adrulipase for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis (CF) has been completed, First Wave BioPharma announced. “We plan to announce the completion of patient enrollment shortly and remain on track to report topline…
The US Food and Drug Administration (FDA) has approved elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta®) for use in patients with cystic fibrosis (CF) aged 2 to 5 years who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that might respond to elexacaftor/tezacaftor/ivacaftor and…
Researchers are developing a protocol that can be used to evaluate interventional trials on the management of pulmonary exacerbations of cystic fibrosis, according to details of a study published in Trials. Pulmonary exacerbations are a hallmark of cystic fibrosis. Around 25% of patients do not fully recover after experiencing one such episode; hence, they are…
A new study has found that the lymphocyte toxicity assay (LTA) may be a useful diagnosis and management tool for drug hypersensitivity reactions (DHRs) in patients with cystic fibrosis (CF). The study, published in Molecular Diagnosis & Therapy, also found evidence that toxic reactive metabolite accumulation might be part of the event cascade leading to…
In children 2 years of age or older with cystic fibrosis (CF), treatment with lumacaftor/ivacaftor (LUM/IVA) early in the disease course may modify disease progression, according to findings from a phase 2, placebo-controlled trial published in Annals of the American Thoracic Society. Among individuals with CF, mutations in the gene that encodes the CF transmembrane conductance…
A study conducted by researchers from Brazil showed that high-throughput sequencing technology (HTS) could be useful to establish a definitive molecular diagnosis of cystic fibrosis (CF). “HTS technology applied to the identification of pathogenic variants associated with the development of this disease allows for the introduction of therapy with CFTR modulators that favour better patient…
Nabriva Therapeutics has announced positive topline results from a phase 1 clinical trial assessing the safety and pharmacokinetics of lefamulin (Xenleta®) in adult patients with cystic fibrosis (CF). In a press release, Christine Guico-Pabia, MD, MBA, MPH, chief medical officer of Nabriva Therapeutics, was thrilled to share the new data. “The data indicate that the…
The treatment of cystic fibrosis (CF) with elexacaftor/tezacaftor/ivacaftor (ETI) might be associated with increased lung function and body mass index (BMI), fewer disease exacerbations, and only mild side effects, according to a study published in the journal Pharmacological Research & Perspectives. This multicenter longitudinal observational study gathered 20 Dutch patients with CF who received ETI…
The addition of specialist palliative care to standard clinic visits for adults with cystic fibrosis (CF) is feasible, acceptable, and efficacious, according to the results of a pilot, single-site, equal-allocation, randomized controlled study published in the Journal of Palliative Medicine. The investigators sought to evaluate the feasibility, acceptability, and perceived efficacy of longitudinal specialist palliative…
A new study has found an association between challenged urine bicarbonate excretion and clinical characteristics in cystic fibrosis (CF). The study, published in the Annals of Internal Medicine, observed partially restored renal cystic fibrosis transmembrane conductance regulator (CFTR) function in patients with CF undergoing therapy with elexacaftor/tezacaftor/ivacaftor. “The objective of this study was to conduct…
