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HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Expand descriptionThis is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
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The Effect of Intra-sinus Application of Betamethasone Dipropionate Nasal Cream on Patients with Chronic Rhinosinusitis Post Functional Endoscopic Sinus Surgery (FESS)
Expand descriptionTo assess the safety and efficacy of one (single) application of Betamethasone Dipropionate Nasal Cream (BMDP CREAM) onto the sinus mucosa.
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A Study to Assess Long-term Safety, Tolerability, and Efficacy of Rocatinlimab in Adult and Adolescent Participants With Moderate-to-severe Atopic Dermatitis (AD)
Expand descriptionThe primary objective of this study is to describe the long-term safety and tolerability of rocatinlimab in participants with moderate-to-severe AD.
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Study of SPG302 in Healthy Volunteers and ALS Participants
Expand descriptionThe first-in-human Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SPG302 in healthy volunteers and ALS participants
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Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
Expand descriptionThis study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (\<) 24 months of age.
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A Study of Retatrutide (LY3437943) in Participants With Obesity and Cardiovascular Disease
Expand descriptionThe main purpose of this study is to evaluate the efficacy and safety of retatrutide once weekly in participants with obesity and established cardiovascular disease (CVD). The study will last about 113 weeks.
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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Expand descriptionThe study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
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A Study to Evaluate the Long-Term Safety of Astegolimab in Participants With Chronic Obstructive Pulmonary Disease (COPD)
Expand descriptionThe purpose of this study is to assess the long-term safety and to explore the efficacy of astegolimab in participants with chronic obstructive pulmonary disease (COPD) who have completed the 52-week placebo-controlled treatment period in parent studies GB43311 or GB44332.
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A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease
Expand descriptionThis study investigates the efficacy and safety of belimumab compared to placebo, in addition to standard therapy, for the treatment of participants with systemic sclerosis associated interstitial lung disease (SSc-ILD). The study will evaluate the effect of belimumab treatment on lung function as well as on extra-pulmonary disease manifestations, including skin thickening and general symptoms, such as fatigue, that impact quality of life (QoL).
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Deep sequencIng in Cutaneous Squamous CEll caRciNomas
Expand descriptionTo comprehensively describe the molecular profile of the tumour ecosystem of cutaneous squamous cell carcinoma (CSCC) patients treated with neoadjuvant immunotherapy using single-cell sequencing and bulk genomic profiling.