A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2

Primary Objectives:

Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period

Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or …

Multicenter Perspective Study: Clinical Evaluation of the Persona MC With Preservation or Sacrifice of the PCL

Primary endpoint

-Evaluation of the survival of the implant after 5 years of follow-up

Secondary endpoints

PROPHET Study: ctDNA-Guided Personalized Induction Immunochemotherapy for NSCLC (PROPHET)

Researchers will use ctDNA dynamics to guide the cycles of induction treatment to see if some patients can avoid excessive cycles of treatment.--UATul>

Intratumoral Tilsotolimod a TLR-9 Agonist Together With Intratumoral Ipilimumab and Intravenous Nivolumab in Patients With Advanced Cancers

The trial will be divided into two parts:

PART A:

the first part will assess the safety of two regimen and will recruit patients with all types of injectable solid malignancies

PART B:

the second part will include 3 expansion cohorts of …

VARA (Virtual and Augmented Reality Applications in Rehabilitation): Motor Rehabilitation Protocol With GRAIL for Patients Affected by Hemiparesis

Study on Biomarkers of Periodontitis and Type 2 Diabetes Mellitus in Males and Females 30 - 70 Years of Age

Participants will be categorized based on point-of-care hemoglobin A1c (HbA1c) and periodontal disease (PD) staging into 9 subgroups. The key objectives of the study are:

A Study to Evaluate the Safety Tolerability and Pharmacokinetics of Orally Inhaled Aerosolized Hydroxychloroquine Sulfate in Healthy Adult Volunteers

Secondary objectives:

To characterize pharmacokinetics (PK) of single dose AHCQ in healthy individuals.

A Study of Subcutaneous Risankizumab Injection for Pediatric Participants With Moderate to Severe Plaque Psoriasis to Assess Change in Disease Symptoms

Part 2: Participants aged 12 < 18 will receive;

A Study on the Safety and Immunogenicity of Influenza, Respiratory Syncytial Virus, Human Metapneumovirus and Parainfluenza Vaccines in Adults 18 to 49 Years of Age.

ul>

Participants will be provided with a diary to solicit reporting of injection site reactions and systemic reactions, unsolicited Adverse Events (AEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest AESIs).

Mobility Protocol Adapted for Advanced Visually Impaired Subjects

People Retinitis Pigmentosa therefore encounter a large number of difficulties in daily life, specifically for:

This study aims to assess the difficulties in the daily life of subjects with …