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Dose-ranging Study to Evaluate the Efficacy, Safety, and Tolerability of AMG 133 in Adult Subjects With Overweight or Obesity, With or Without Type 2 Diabetes Mellitus
Expand descriptionThe study aims to compare and assess the dose response of 3 selected doses of maridebart cafraglutide compared with placebo, on inducing and maintaining weight loss from baseline at Week 52 in participants with overweight or obesity without diabetes mellitus (Cohort A) and in participants with overweight or obesity with Type 2 diabetes mellitus (Cohort B).
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A Study of DZD9008 Versus Platinum-Based Doublet Chemotherapy in Local Advanced or Metastatic Non-small Cell Lung Cancer (WU-KONG28)
Expand descriptionThis is a phase 3, open-label, randomized, multi-center study assessing the efficacy and safety of DZD9008 versus platinum-based doublet chemotherapy in participants with locally advanced or metastatic NSCLC with EGFR Exon20ins mutation, who are newly diagnosed or have not received prior systemic therapy in advanced stage. Primary objective of this study is to assess the efficacy of DZD9008 versus platinum-based doublet chemotherapy using by BICR-assessed PFS per RECIST 1.1 as primary endpoint. Approximately 320 participants are estimated to be randomized into the study. Participants enrolled will be randomized to DZD9008 or platinum-based doublet chemotherapy in a 1:1 manner, stratified by baseline brain metastasis (with/without).
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A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Expand descriptionThe purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
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Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children (MARVEL-PIC)
Expand descriptionA prospective, open, randomised implementation study in paediatric cancer patients. The study aims to determine whether a personalised approach will result in an overall reduction in clinically relevant adverse drug reactions (ADRs) and to evaluate the economic and quality of life impacts. Participants will be randomised to receive personalised guided prescribing of supportive care therapy (study arm) or standard of care (control arm) for a period of 12 weeks. The follow up period includes prospective patient reporting of symptoms and quality of life through electronically delivered surveys, for a maximum of 12 months.
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Safety, Tolerability and Immunogenicity of an Inactivated Whole-cell Pneumococcal Vaccine Gamma-PN3.
Expand descriptionThis is a randomised placebo-controlled first-in-man dose-ranging study to determine safety and markers of efficacy.
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Pharmacokinetics and Pharmacodynamics Study of LBS-008 in Healthy Volunteers Aged 50-85
Expand descriptionThis is a Phase 1b, parallel single-dose study to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of Tinlarebant when administered as an oral dose to elderly healthy volunteers. This study will evaluate 2 dose levels in 2 cohorts comprising up to a total of 16 participants (8 per cohort). Dose levels will be evaluated in parallel.
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Study to Evaluate Itraconazole Administered as Inhaled Dry Powder in Adults With Asthma and ABPA
Expand descriptionThe goal of this clinical trial is to learn about PUR1900 as an inhaled, antifungal therapeutic for the treatment of allergic bronchopulmonary aspergillosis (ABPA) in patients with asthma. The main questions it aims to answer are: 1. Is PUR1900 safe and well tolerated in adults with asthma and ABPA? 2. Is there an effect of daily administration of PUR1900 on potential outcome measures in adults with asthma and ABPA? 3. Is there fungal resistance to A. fumigatus? This study includes a 28-day screening period, a 112-day (16-week) treatment period, and a 56-day (8 week) observation period. Participants will take either 40mg of PUR1900, 20 mg of PUR1900 or Placebo for 112 days and complete an eDairy, answer questions about their asthma and complete peak respiratory flow measurements at home. They will come to the clinic approximate once a month during the treatment period and complete study assessments. At the end of the observation period participants will complete one more clinic visit. Participants who complete this study may be given the opportunity to continue on study drug in an open label extension study.
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An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Expand descriptionThe purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
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A Study to Evaluate XEN1101 as Adjunctive Therapy in Primary Generalized Tonic-Clonic Seizures
Expand descriptionThis is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the clinical efficacy, safety, and tolerability of XEN1101 administered as adjunctive treatment in primary generalized tonic-clonic seizures (PGTCS).
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CAR-T Cell Therapy in RelApsed/Refractory Myeloma With ExtrameduLlary Disease - an in Vivo Imaging and Molecular Monitoring Study
Expand descriptionThis clinical trial will investigate the in vivo trafficking of cilta-cel in extramedullary myeloma using 64Cu Super Paramagnetic Iron Oxide Nanoparticle (64Cu SPION) and Positron Emission Tomography-Magnetic Resonance Imaging (PET-MRI)