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A Phase II, Randomized, Double-Blind, Placebo- Controlled Study of Ensifentrine in Subjects With Non-Cystic Fibrosis Bronchiectasis
This study is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ensifentrine inhalation suspension (3 mg) deli…
A Pilot Study of Chemoprevention With Tamoxifen in Patients With Pre-Invasive Pancreas Mucinous Cystic Neoplasms Who Will Not Undergo Immediate Resection
Pancreatic mucinous cystic neoplasm (MCN) is a precursor to invasive pancreatic adenocarcinoma which occurs almost exclusively in females in their 5th-7th deca…
Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
This clinical trial is examining the action and effects of several new drugs in the treatment of cystic fibrosis in children. In addition, several genetic fact…
Sinus Disease in Young Children With Cystic Fibrosis
This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on ch…
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…
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 (…
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…
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…
Skin-interfaced Colorimetric Bifluidic Sweat Sensor Device for the Diagnosis of Cystic Fibrosis (CF)
Cystic fibrosis (CF) is a multisystem autosomal recessive inherited disease affecting approximately 75,000 individuals in USA. The sweat chloride (Cl) test rem…