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E.N.D.E.A.V.O.R.: An Exemestane Needed Dose Efficacy and Verification as an Ovulation Induction Regimen Study
The goal of this clinical trial is to learn if study drug exemestane will increase chances of ovulation for patients with polyendocrine metabolic ovarian syndr…
A Phase 1, Open-label, Randomized, Pilot and Feasibility Trial to Evaluate the Safety and Tolerability of the Achromobacter-targeting Bacteriophage Cocktail, AchromoPhage, Among Persons With Cystic Fibrosis and Chronic Achromobacter Lung Infections: The AchromoPhage Trial
The goal of this clinical trial is to learn if a new treatment called AchromoPhage is safe and well tolerated in adults with cystic fibrosis (CF) who have long…
A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
Natural History of Bronchiectasis
Background: * Bronchiectasis is a disease characterized by airways that are inflamed, abnormally dilated, and chronically infected. Individuals with bronchiec…
Rethinking Early Airway Clearence Therapies
The REACT trial consists of two parallel, randomized studies; the Hypertonic Saline Study and the Dornase Alfa Study. Health outcomes among people with cystic…
The Effect of Verapamil on Beta Cell Function in Adolescents and Adults With Cystic Fibrosis-related Dysglycemia
The investigators are conducting a pilot open-label pre/post interventional trial in adolescents and adults with cystic fibrosis (CF) and abnormal glucose tole…
Standardizing Treatments for Pulmonary Exacerbations: A Platform for Evaluating Treatment Decisions to Improve Outcomes (STOP360) Aminoglycoside Study (AG Study)
The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tu…
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) mo…
DICER1-Related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study
Background: \- Pleuropulmonary blastoma (PPB) is a rare fast-growing lung tumor that is associated with other, rare tumor types. Most cases of PPB appear in c…
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
A Phase Ic, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, and Activity of Inhaled GDC-6988 in Patients With Muco-obstructive Disease
This study evaluates the safety, tolerability, and activity of inhaled GDC-6988 in participants with muco-obstructive disease.
TIDES 2.0: Prevalence and Longitudinal Course of Depression, Anxiety, and Behavior Problems in Children With Cystic Fibrosis Under 12 Years of Age
This is a longitudinal, observational epidemiological study designed to estimate the prevalence of depression, anxiety, and behavior problems in children ages …