Assistance Publique Hôpitaux De Paris, France

A Study of DZD9008 Versus Platinum-Based Doublet Chemotherapy in Local Advanced or Metastatic Non-small Cell Lung Cancer (WU-KONG28)

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

CAR-T Cell Therapy in RelApsed/Refractory Myeloma With ExtrameduLlary Disease - an in Vivo Imaging and Molecular Monitoring Study

This a Phase Ib exploratory study designed to investigate the in vivo trafficking of cilta-cel in extramedullary myeloma (EMM) using 64Cu SPION nanoparticles and PET-MRI imaging. It is planned that 10-30% of clinical dose of target number of cilta-cel will be labelled. The target number cilta-cel has been chosen based on the previous first in humans (FIH) study. The rationale to label of cilta-cel in the range of ≤30% is to ensure that reasonable positron emission tomography (PET) and magnetic resonance (MR) imaging quality by increasing the relative labelling dose, in the case low cell numbers are obtained. Additionally, the selected range is chosen to limit cellular toxicity and radiation exposure to the patient from the labelled cells. The unlabeled and labelled dose will be administered as scheduled by a two-part intravenous infusion in which the labelled cells are administered no later than 4hrs after the unlabeled infusion

Safety and Tolerability of a Novel Implantable Device for the Treatment of Erectile Dysfunction

Metastatic Leiomyosarcoma Biomarker Protocol

4D-310 in Adults With Fabry Disease and Cardiac Involvement

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement

Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease

Non-invasive ventilation (NIV) is a treatment that uses positive pressure delivered via a face mask or mouthpiece to assist a person to breathe. It can be used as a long-term treatment for people whose breathing is failing - usually due to chronic conditions that produce weakness of the respiratory muscles such as motor neurone disease / amyotrophic lateral sclerosis [MND/ALS]chronic obstructive pulmonary disease). Most people with MND/ALS use NIV at night initially. Even though NIV may improve survival and function, many are unable to use it for more than 4 hours per day (which is considered a threshold amount of use in order to gain a benefit) and many others are unable to tolerate it at all. Our team has recently provided evidence that specific and individualised titration of NIV leads to better outcomes in people with MND. This previous trial determined that the use of a sleep study (also called 'polysomnography') can improve the way people are initially set up with NIV. This study will replicate and extend the single site study in a large, multi-centre randomised controlled trial (RCT) across multiple sites This multi-centre RCT will also include a 12-month follow-up period to evaluate longer-term outcomes.

A Study of CLN-619 Alone and in Combination With Pembrolizumab in Advanced Solid Tumors

A Study of NVL-520 in Patients With Advanced NSCLC and Other Solid Tumors Harboring ROS1 Rearrangement (ARROS-1)

In Phase 2, study patients will be enrolled into 5 distinct expansion cohorts: - Cohort 2a: ROS1-positive NSCLC naïve to Tyrosine Kinase Inhibitor (TKI) therapy and up to 1 prior chemotherapy and/or immunotherapy. - Cohort 2b: ROS1-positive NSCLC treated with 1 prior ROS1 TKI and no prior chemotherapy or immunotherapy. - Cohort 2c: ROS1-positive NSCLC treated with 1 prior ROS1 TKI and 1 prior platinum-based chemotherapy with or without immunotherapy. - Cohort 2d: ROS1-positive NSCLC treated with ≥2 prior ROS1 TKIs and up to 1 prior chemotherapy and/or immunotherapy. - Cohort 2e: ROS1-positive solid tumor and progressed on any prior therapy.