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Cystic Fibrosis clinical trials at UCSD

7 in progress, 3 open to eligible people

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  • PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis

    open to eligible people ages 6-99

    Isolation of nontuberculous mycobacteria (NTM) from the sputum of individuals with CF is an increasingly common finding, and the lack of an evidenced-based approach to treatment of NTM disease has been identified as one of the greatest unmet needs within the CF community. Current evidence predicts that the prevalence of NTM will remain relatively high in the CF population. Approaches to NTM disease treatment differ widely between centers, and expected outcomes are not known. This study is observational and follows current best practices. The study will help define response to treatment, and collect relevant data associated with treatment of NTM disease to build a framework for future therapeutic trials.

    San Diego, California and other locations

  • PREDICT Trial: PRospective Evaluation of NTM Disease In CysTic Fibrosis

    open to eligible people ages 6-99

    Isolation of nontuberculous mycobacteria (NTM) from the sputum of individuals with CF is an increasingly common finding, and the lack of an evidenced-based approach to the diagnosis of NTM disease has been identified as one of the greatest unmet needs within the CF community. Current evidence predicts that the prevalence of NTM will remain relatively high in the CF population. Approaches to NTM disease diagnosis differ widely between centers. This study is observational and follows current best practices. The study will help standardize the diagnosis and collect relevant data associated with the diagnosis of NTM disease to build a framework for future therapeutic trials.

    San Diego, California and other locations

  • Prospective Study of Pregnancy in Women With Cystic Fibrosis

    open to eligible females ages 16 years and up

    In this study, the investigators aim to evaluate changes in lung function in women with cystic fibrosis (CF) during pregnancy and for 2 years after pregnancy based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

    La Jolla, California and other locations

  • A Phase 1b/2 Trial of the Safety and Microbiological Activity of Bacteriophage Therapy in Cystic Fibrosis Subjects Colonized With Pseudomonas Aeruginosa

    Sorry, not yet accepting patients

    This is a phase 1b/2 study of a single dose of intravenous (IV) bacteriophage in males and non-pregnant females, at least 18 years old and diagnosed with Cystic Fibrosis (CF), and residing in the United States. This clinical trial is designed to assess the safety and microbiologic activity of bacteriophage product WRAIR_PAM-CF1, directed at Pseudomonas aeruginosa in clinically stable CF individuals. WRAIR_PAM-CF1 is a 4 component anti-pseudomonal bacteriophage containing between >/= 4 x 107 and >/= 4 x 109 Plaque Forming Units (PFU) in the target dose. Enrollment will occur at up to 20 clinical research sites in the United States. In stage 1, two eligible subjects will be assigned to each of the three dosing arms receiving a single dosage of the IV bacteriophage therapy (4 x 107 PFU, 4 x 108 PFU, and 4 x 109 PFU; total of 6 sentinel subjects), followed by a 96-hour observation period. Stage 2a will proceed if no serious adverse events (SAEs) related to the study product occur during the observation period of stage 1; 32 subjects will be enrolled into one of 4 arms (placebo IV, 4 x 107 PFU, 4 x 108 PFU, and 4 x 109 PFU) in a 1:1:1:1 allocation. An interim analysis after all subjects have completed follow up visit 5 on Day 30 will be performed to select an IV bacteriophage dose with the most favorable safety and microbiologic activity profile. During Stage 2b, subjects will be randomized into the bacteriophage (dose selected based on Interim Analysis following Stage 2a) or placebo arm. The final sample size is expected to be up to 72 subjects total with up to 25 subjects in the placebo arm and up to 25 subjects in the Stage 2b bacteriophage dose.

    La Jolla, California and other locations

  • Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis

    Sorry, in progress, not accepting new patients

    The long-term goal is to study the safety and effectiveness of hormonal contraception for women with cystic fibrosis (CF) and contribute to national guidelines that the Cystic Fibrosis Foundation and the Centers for Disease Control and Prevention (CDC) provide to clinicians. The study objectives are to determine whether hormonal contraceptive methods improve overall pulmonary health, worsen CF-related disease or CF liver disease, or are effective against unwanted pregnancy with concomitant CF transmembrane conductance regulator (CFTR) modulator use. The hypothesis is that hormonal contraceptive methods are safe and do not worsen CF-related complications over time,improve FEV-1 when compared to non-hormonal users, and oral birth control methods with CFTR modulator use.

    San Diego, California and other locations

  • CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

    Sorry, in progress, not accepting new patients

    This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and currently receiving commercially approved Cystic Fibrosis transmembrane conductance regulator (CFTR) modulator therapies.

    La Jolla, California and other locations

  • Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy

    Sorry, in progress, not accepting new patients

    Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating CF, it is still largely unknown whether or not other chronic therapies can be safely stopped. The SIMPLIFY study is being done to test whether or not it is safe to stop taking inhaled hypertonic saline or Pulmozyme® (dornase alfa) in those people that are also taking Trikafta™. Trikafta (elexacaftor/tezacaftor/ivacaftor) is a combination CFTR modulator therapy that was approved by the Food and Drug Administration for people with CF who have at least one F508del mutation. The three drugs that make up Trikafta work together to allow many more chloride ions to move into and out of the cells, improving the balance of salt and water in the lungs. These changes result in better clearance of mucus from the lungs and improvements in lung function. Inhaled hypertonic saline and Pulmozyme (dornase alfa) also improve clearance of mucus from the lungs to support lung function and have been available to people with CF for many years. Both therapies are considered to be relatively burdensome and it is not known whether either therapy can improve or maintain lung function above what is already gained through Trikafta use. The goal of the SIMPLIFY study is to get information about whether or not it is safe to stop either inhaled hypertonic saline or Pulmozyme (dornase alfa) by testing if there is a change in lung function in subjects with cystic fibrosis (CF) who are assigned to stop their chronic medication (either hypertonic saline or Pulmozyme) as compared to those who are assigned to keep taking their medication while continuing to take Trikafta.

    San Diego, California and other locations

Our lead scientists for Cystic Fibrosis research studies include .

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