View clinical trials related to Pulmonary Alveolar Proteinosis.
Filter by:This is a randomized, double-blind, placebo-controlled, single ascending (SAD), and multiple ascending dose (MAD) study conducted at a single clinical site within the UK. Healthy male and female subjects (on non-child bearing potential) will be enrolled to investigate single inhaled doses of molgramostim at 3 dose levels (Part 1) and multiple inhaled doses at 2 dose levels (Part 2). The 2 doses in the multiple ascending dose regimens will be administered once daily (QD) for 6 consecutive days. The clinical indication for inhaled molgramostim is the treatment of respiratory diseases such as aPAP, bronchiectasis and cystic fibrosis. The Clinical trial will involve 42 healthy participants. The trial is expected to last approximately 4 months.
The purpose of this study is twofold: to assess routine cat scan (CT) imaging as a biomarker for removal of lipoprotein surfactant via lung lavage (where CT is the gold-standard imaging technique for density quantification), and to utilize a novel UTE MRI protocol to similarly quantify surfactant removal. This study will also serve to generate baseline scanning that may aid in developing analytical tools to evaluate and treat specific lung regions of patients with PAP.
Background: - Healthy people have white blood cells that protect them against bacteria, viruses, and fungi. However, some people have diseases which cause the body to make white blood cells that do not work properly. These white blood cells can attack the body s own proteins. These types of diseases are called anti-cytokine autoantibody-associated diseases. They can cause severe illnesses and even death. They are also difficult to treat with standard drugs. - Rituximab is a drug used to treat rheumatoid arthritis. It attacks white blood cells that do not work properly. Currently, it is not approved for treating anti-cytokine autoantibody-associated diseases. However, researchers think that it may be able to help treat people with these immune diseases. Objectives: - To see if rituximab is a safe and effective treatment for anti-cytokine autoantibody-associated diseases. Eligibility: - Individuals at least 18 years of age who have anti-cytokine autoantibody-associated diseases. - Participants must also be enrolled in a related immune disorder study at the National Institutes of Health. Design: - The study will last 24 months. Participants will take rituximab for 6 months and have follow-up visits for the remaining 18 months. - Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Other samples will be collected as needed if participants currently have an infection. - Participants will enter the hospital for 1 week at the start of treatment. They will have four doses of rituximab given 2 days apart. This first treatment will be monitored with frequent blood tests. - Over the next 6 months, participants will have four more doses of rituximab given about 1 month apart. Treatment will be monitored with frequent blood tests and sample collections as needed. - There will be four follow-up study visits at 3, 6, 12, and 18 months after the last dose of rituximab.
The purpose of this study is to evaluate the therapeutic efficacy of inhaled recombinant human GM-CSF in individuals with hereditary Pulmonary Alveolar Proteinosis (PAP) due to partial dysfunction of the GM-CSF receptor.
This is a prospective, randomized, open-label, long-term, phase 2 study of inhaled granulocyte/macrophage-colony stimulating factor following whole lung lavage therapy in patients with autoimmune pulmonary alveolar proteinosis.
The purpose of this study is to determine whether the use of rituximab is effective in treating pulmonary alveolar proteinosis by leading to an improvement in lung function and disease status.