Muscular Dystrophy Clinical Trials

Effect of GLP-1 Agonist Therapy on Insulin Secretion in Adults with Pancreatic Insufficient Cystic Fibrosis and Abnormal Glucose Tolerance: a randomized open-label cross-over trial

Diabetes is a major co-morbidity in pancreatic insufficient cystic fibrosis (PI-CF) and associated with worse outcomes. While reduced -cell mass contributes to the insulin secretory defects that characterizes cystic fibrosis-related diabetes (CFRD), other modifiable determinants appear operative in the emergence and progression of abnormal glucose tolerance towards diabetes. Identifying interventions …

Observational Study Protocol Chart Review of the Clinical Outcomes in Hepatorenal Syndrome in the United States

TBD

A Randomized Double-Blinded Placebo-Controlled Trial to Investigate the Efficacy Safety and Tolerability of Efgartigimod PH20 SC in Adult Patients With Pemphigus (Vulgaris or Foliaceus) ARGX-113-1904

4.0

An Open-Label Multicenter Follow-up Trial of ARGX-113-1904 to Evaluate the Safety Tolerability and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus ARGX-113-1905

4.0

An extended access program to assess long-term safety of bardoxolone methyl in patients with pulmonary hypertension

This is a Phase 3a, extended access program to assess long-term safety of bardoxolone methyl in patients with pulmonary hypertension. The purpose of this study is to provide or continue to provide bardoxolone methyl to eligible subjects who participated in the CATALYST study. The study will also collect ongoing safety …

A PHASE II RANDOMIZED TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF FECAL MICROBIOTA TRANSPLANTATION USING THE PENN MICROBIOME THERAPY PRODUCTS FOR SEVERE OR SEVERE-COMPLICATED/FULMINANT CLOSTRIDIUM DIFFICILE INFECTION

A PHASE II RANDOMIZED TRIAL TO EVALUATE THE OPTIMAL DOSING OF FECAL MICROBIOTA TRANSPLANTATION USING THE PENN MICROBIOME THERAPY PRODUCTS FOR RECURRENT CLOSTRIDIUM DIFFICILE INFECTION

Please reference attached protocol document, section "Study Summary" located on pages 7,8,9

An open-label multi-center roll-over study to assess long

The purpose is to evaluate the long-term safety of osilodrostat in patients who have already received osilodrostat treatment in a previous Novartis trial for treatment of Cushing's syndrome.

A Study to Evaluate the Safety and Efficacy of Image Guided Surgery using Indocyanine Green for Intramolecular Imaging of Solid Tumors Compared to Standard of Care (TumorGlow) (PRIME)

Indocyanine Green (ICG) is an FDA approved, water-soluble tricarbocyanine dye routinely used in clinical settings for measuring cardiac output, liver function, and retinal angiography and has been in use for over 50 years. Patient diagnosed with solid tumor cancers have a high rate of recurrence when surgery is a treatment. …

The HepQuant SHUNT Liver Diagnostic Kit for Likelihood of Large Esophageal Varices: The SHUNT-V Study

The purpose of the HepQuant SHUNT study is to validate the Disease Severity Index (DSI) from the HepQuant SHUNT Test for likelihood of large esophageal varices by comparing the SHUNT test to the current standard of care, EGD, in patients with chronic liver disease.