Multicohort Trial of Different Schemes of PM14 in Monotherapy and in Combination With Radiotherapy in Soft Tissue Sarcomas and Other Solid Tumor

Phase Ib/II, multicohort, single arm, open-label, multicenter, international clinical trial, with 6 cohorts (advanced STS, advanced L-sarcomas, other advanced sarcomas, advanced solid tumors, and localized STS) with 4 sites in Spain for phase I. The aim of this study is to explore different infusions of PM14 (longer or repeated) in …

J Joanna Kwiatkowska, MD, PhD

The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD

The study includes 150 patients with DMD diagnosis confirmed by genetic testing, 8-16 years old (≥8 and <17) at the study entry with a follow-up of up to 5 years. Random enrollment of a patient to one of two groups (intervention or control) takes place after pre-screening and screening stage …

S Sanne V. Hovgesen, MD

Lithium Versus Cariprazine in the Acute Phase Treatment of Bipolar Depression (DUAG9)

The goal is to study the effect of lithium compared to cariprazine in patients with depression in a bipolar disease. The main question it aims to answer is: Difference in change between the two groups from baseline to after 8 weeks treatment on Hamilton Ratings Scale for Depression, 6-item version …

B Bethany Raiff, PhD

Re-Connect Application for Smoking Cessation

This series of studies will explore the acceptability, feasibility, and preliminary efficacy of making access to smartphone applications contingent on objective evidence of smoking abstinence.

C CHAOUAT ARI, MD, PHD

Study of Cardiac MRI in the Follow up Assessment of Patients With PAH (EVITA)

Pulmonary arterial hypertension (PAH) is characterized by a progressive increase in pulmonary vascular resistance leading to right ventricular failure and eventually to death. The therapeutic strategy has become complex and needs to perform recurring follow up evaluations including right heart catheterizations (RHC). Cardiac magnetic resonance imaging (cMRI) has the advantage …

Effects of Acute and Chronic Empagliflozin Heart Failure

This is a 60-patient randomized, double-blind, placebo-controlled mechanistic study to understand the utility of empagliflozin in worsening heart failure (HF) patients with or without diabetes. Participants will be randomized to empagliflozin or placebo for 6 weeks, followed by a crossover of placebo patients to active therapy at 6 weeks-12 weeks.

R Ryan Nelson, DPT

ACL Bone Health and Loading Study

The purpose of this study is to look at the effects of bone density on lower extremity loading mechanics using Blood Flow Restriction (BFR) to provide the control group for bone health. Hypothesis: The BFR group will have decreased bone mineral loss and improved functional performance compared to control group.

E Emma Canepa, MS, CCRP

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

A AJ Smits, MD

ORthosis vs No Orthosis After Surgically Treated Traumatic Thoracolumbar Fractures

Rationale: There is no evidence in the current literature regarding the additional value of an orthosis after surgically treated thoracolumbar spine fractures. Objective: To assess whether an orthosis provides additional pain relief compared to no orthosis after posteriorly fixated thoracolumbar spine fractures. Primary outcome is difference in pain at six …

S Scott K Cherne, MD

Safety and Effectiveness of the PXL-Platinum 330 System for Corneal Collagen Cross-Linking in Eyes With Corneal Thinning Conditions

To determine whether the Peschke PXL-330 system is safe and effective in the treatment of corneal thinning conditions.