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A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine p…
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Controlled Trial (STOP PEDS RCT)
The STOP PEDS RCT is a multicenter, parallel, open label randomized controlled trial evaluating the long-term (one year) and short-term safety and efficacy of …
Rare CFTR Mutation Cell Collection Protocol (RARE)
Over 1,900 mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein are implicated in causing Cystic Fibrosis (CF). Pot…
A Phase 2a, Randomized, Placebo-Controlled, Double Blind Multiple Ascending Dose Study in Patients With Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat w…
A Phase I Randomised, Single-blind, Placebo-controlled Study to Assess the Safety, Tolerability, and Pharmacokinetics of AZD1613 Following Multiple Ascending Dose Administration in Participants With Autosomal Dominant Polycystic Kidney Disease
A study to investigate safety, tolerability, and pharmacokinetics of AZD1613 following subcutaneous or intravenous administration in participants with autosoma…
Estrogen Supplementation and Bone Health in Women With CF (STURDY)
The goal of this study is to learn about the role of estrogen and other hormones in bone development in adolescent and young adult women with cystic fibrosis (…
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…
Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions. Stan…
Implementing Digital Retinal Exams Into Comprehensive Pediatric Diabetes Care
The purpose of this study is to determine if use of a nonmydriatic fundus camera using autonomous artificial intelligence software at the point of care increas…
A Phase 1 Dose Escalation and Expansion Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 in Subjects With Advanced and/or Metastatic Solid Tumors Known to Express c-Kit
This open-label clinical trial will evaluate the safety and tolerability of NN3201 in subjects with advanced and/or metastatic solid tumors known to express c-…
CGM Dynamic Index (CDI) for Predicting Prediabetes in People With Cystic Fibrosis
The primary objective of this pilot study is to develop a CGM-based model to predict the progression from prediabetes to diabetes in individuals with cystic fi…
Sinonasal Therapies and Histologic Correlations for Patients With Cystic Fibrosis in the Era of Highly Effective Modulator Therapy
The investigators are doing this study to discover if it is appropriate for people with Cystic Fibrosis (CF) on highly effective modulator therapy (HEMT) to st…