Muscular Dystrophy Clinical Trials

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 3 Randomized Comparator-Controlled Clinical Trial to Study the Efficacy of Pembrolizumab (MK-3475) in Combination with Bacillus Calmette-Guerin (BCG) in Participants with High Risk Non-muscle Invasive Bladder Cancer (NMIBC) that is Persistent or Recurrent Following BCG Induction (KEYNOTE-676)

This study will involve patients with high risk non-muscle invasive bladder cancer (NMIBC) that is persistent (cancer stays) or recurrent (cancer comes back) following Bacillus Calmette-Guérin BCG treatment. The purpose of this study is to: o Test the safety of the study drugs, pembrolizumab in combination with Bacillus Calmette-Guerin (BCG) …

A first in human phase 1 study in healthy volunteers and heterozygous carriers of a loss-of-function Granulin mutation causative of frontotemporal dementia to assess AL001 safety tolerability pharmacokinetics and pharmacodynamics

Please see section 1 of protocol

A Phase 2 Multicenter Open-Label Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia

Please see section 1 of protocol

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. …

A Pilot Study of Diffusion Weighted MRI to Assess Esophageal Tumor Response to Neoadjuvant Chemoradiation

The purpose of this study is to determine if a particular Magnetic Resonance Imaging (MRI) called Diffusion Weighted (dwMRI)) will detect changes in your tumor before and during treatment with chemotherapy and radiation for esophageal cancer. We used the dwMRI because it has been used in other types of cancers …