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Clinical Trial of Oral Digoxin In NASH (CODIN)
Nonalcoholic steatohepatitis (NASH) is a severe subtype of nonalcoholic fatty liver disease (NAFLD) which affects 1 in 3 Americans. The mainstay of treatment f…
Natural History of Noncirrhotic Portal Hypertension
Background: \- Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start…
A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis
This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of P…
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…
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…
RALLY-MF: A Phase 1b/2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of DISC-0974 in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
This phase 1b/2a open-label study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974 as well as categorize the effects on…
A Phase 3 Study to Evaluate the Efficacy and Safety of Pegozafermin in Subjects With Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis
The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adul…
Therapeutic RSK1 Targeting in Myelofibrosis
This is a phase Ib study evaluating PMD-026, an oral inhibitor of ribosomal protein S6 kinase A1 (RSK1), in participants with myelofibrosis (MF).The dose escal…
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…
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.
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 Study of the Natural Progression of Interstitial Lung Disease (ILD)
We propose to acquire data and blood samples on all patients being cared for by the Interstitial Lung Disease (ILD) program. Additionally, we will collect data…