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A Pilot Study to Evaluate the Safety and Tolerability of Baricitinib in Patients With Job s Syndrome With Lupus-like Disease and/or Atopic Dermatitis
Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin an…
A Phase IIb Randomized, Double-blind, Placebo-controlled, Parallel, Multidose Study to Evaluate the Efficacy, Safety, and PK of AZD0292 in Participants 12 Years of Age and Older With Bronchiectasis and Chronic Pseudomonas Aeruginosa Colonization
AZD0292 is a bispecific IgG1k mAb being evaluated for the prevention of exacerbations in bronchiectasis patients chronically colonized with PsA.
Clinical Genetics Branch (CGB) Eligibility Screening Survey
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding …
Natural History of Bronchiectasis
Background: * Bronchiectasis is a disease characterized by airways that are inflamed, abnormally dilated, and chronically infected. Individuals with bronchiec…
A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Investigate Efficacy, Safety, Immunogenicity, and Pharmacokinetics, of GSK3862995B in Participants With Bronchiectasis
This study is designed to find out how effective and safe a new drug, GSK3862995B, is for adult participants with bronchiectasis, a chronic lung disease. The s…
Evaluation of Ventilation Defects Downstream of Mucus Plugs in Patients With Muco-Obstructive Lung Disease
In this study, xenon MRI will be used to evaluate regional functional consequences of mucus plugs in the lungs of patients with muco-obstructive pulmonary dise…
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like subs…
A Phase 1b, Open-label, Multi-cohort Study of AZD0120, an Autologous CD19/BCMA Targeting Chimeric Antigen Receptor T-cell, in Adults With Autoimmune Diseases
This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and toler…
Neurologic Bone Marrow Derived Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3…
An Open-label, Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 Following Single-ascending Dose and Step-up Dose Administration to Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies or Rheumatoid Arthritis
The purpose of this study is to measure the safety, tolerability, PK, and PD of AZD5492 administered subcutaneously in adult participants with SLE or IIM or RA…
A Phase III, Randomised, Double-blind, Placebo-controlled Study to Assess the Efficacy, Safety, and Tolerability of BI 1291583 2.5 mg Administered Once Daily for up to 76 Weeks in Patients With Bronchiectasis (The AIRTIVITY™ Study)
This study is open to adults and adolescents aged 12 to under 18 with bronchiectasis. People can participate in this study if they produce sputum and have had …
Electrical Impedance Myography: Natural History Studies in Neuromuscular Disorders and Healthy Volunteers
Background: \- Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorder…