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Pulmonary Alveolar Proteinosis clinical trials

View clinical trials related to Pulmonary Alveolar Proteinosis.

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NCT ID: NCT06111846 Not yet recruiting - Clinical trials for Autoimmune Pulmonary Alveolar Proteinosis

Study of Human Bone Marrow Mesenchymal Stem Cells in aPAP

Start date: November 1, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this open-label phase IIa clinical trial study is to evaluate the safety and preliminary efficacy of hBMMSC intravenous treatment in patients with aPAP.

NCT ID: NCT05761899 Recruiting - Clinical trials for Hereditary Pulmonary Alveolar Proteinosis

Safety and Efficacy of PMT Therapy of hPAP

Start date: June 26, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

The major goal of this study is to evaluate a new type of cell transplantation therapy for individuals with hereditary PAP, study a new treatment that may be useful for treatment of other diseases, and research mechanisms that drive the development and function of lung macrophages.

NCT ID: NCT05640687 Active, not recruiting - Clinical trials for Pulmonary Alveolar Proteinosis(PAP)

The Study of Mesenchymal Stem Cells Treat Autoimmune Pulmonary Alveolar Proteinosis in Vitro

Start date: March 1, 2021
Phase:
Study type: Observational

Autoimmune pulmonary alveolar proteinosis (aPAP) is a respiratory disease characterized by massive deposition of pulmonary surfactant in the alveoli, involving a variety of immune cells and factor disorders. However, there are certain limits in treatment at present. MSCs can improve the microenvironment in the alveoli by regulating immunity, thereby achieving a good therapeutic effect. The purpose of this study is to use the lavage fluid obtained after whole lung lavage with aPAP to isolate alveolar macrophages, and to use MSC to complete the verification of the efficacy of aPAP primary alveolar macrophages in vitro. A series of protocols including multi-factor detection, cell phenotype analysis and phagocytosis assay were used to evaluate the efficacy of MSCs on alveolar macrophages.

NCT ID: NCT05300360 Enrolling by invitation - Clinical trials for Pulmonary Alveolar Proteinosis

Prevalence of Adenosine Deaminase (ADA) Enzyme Deficiency Disease in Adult Patients With Pulmonary Alveolar Proteinosis

Start date: August 16, 2021
Phase:
Study type: Observational

This observational study was designed as a prospective epidemiological screening study. Patients who have applied to the centers participating in the study and who have previously been clinically or pathologically diagnosed with PAP (Pulmonary alveolar proteinosis) will be included in the study. Up-to-date data will be collected from patients who have agreed to participate in the study, and a blood sample with DBS will be taken from patients. The blood taken will be subjected to analysis for ADA metabolites. For patients with a high metabolic test, the responsible researcher will advise on clarifying the diagnosis with a genetic test other than the study. In case of formation of new information for each patient, consultation will be provided by the responsible researcher. Thus, the prevalence of ADA enzyme deficiency disease will be evaluated in patients diagnosed with PAP.

NCT ID: NCT04811274 Recruiting - Genetic Mutation Clinical Trials

Macrophages, GM-CSF and MARS Proteinosis

MacroMARS
Start date: June 7, 2021
Phase:
Study type: Observational

Mutations in the MARS gene encoding methionyl-tRNA synthetase are responsible for a genetic form of alveolar proteinosis (PAP), but the pathophysiological mechanisms of the respiratory phenotype are not known. The main hypothesis is that the PAP phenotype in these patients is secondary to a defective clearance of the surfactant by the alveolar macrophages. The main objective of the study is to study the clearance capacity of lipoproteinaceous material by macrophages of patients with MARS related PAP. This will be investigate in cultured macrophages derived from peripheral blood monocytes of patients (patients with MARS related PAP) and controls (patients without MARS related PAP).

NCT ID: NCT04544293 Active, not recruiting - Clinical trials for Autoimmune Pulmonary Alveolar Proteinosis

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

IMPALA-2
Start date: May 10, 2021
Phase: Phase 3
Study type: Interventional

160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim or placebo for 48 weeks. Subjects completing the 48 week placebo-controlled period will receive open-label treatment with once daily inhaled molgramostim for 96 weeks.

NCT ID: NCT04516577 Completed - Clinical trials for Pulmonary Alveolar Proteinosis

Updated Severity and Prognosis Score of Pulmonary Alveolar Proteinosis

Start date: January 1, 2020
Phase:
Study type: Observational

By updating the chest HRCT scoring criteria of patients with pulmonary alveolar proteinosis, a new and more perfect system for evaluating the severity of alveolar proteinosis will be established.

NCT ID: NCT04326036 Enrolling by invitation - COPD Clinical Trials

Use of cSVF Via IV Deployment for Residual Lung Damage After Symptomatic COVID-19 Infection

GARM-COVID19
Start date: March 25, 2020
Phase: Early Phase 1
Study type: Interventional

COVID-19 Viral Global Pandemic resulting in post-infection pulmonary damage, including Fibrotic Lung Disease due to inflammatory and reactive protein secretions damaging pulmonary alveolar structure and functionality. A short review includes: - Early December, 2019 - A pneumonia of unknown cause was detected in Wuhan, China, and was reported to the World Health Organization (WHO) Country Office. - January 30th, 2020 - The outbreak was declared a Public Health Emergency of International Concern. - February 7th, 2020 - 34-year-old Ophthalmologist who first identified a SARS-like coronavirus) dies from the same virus. - February 11th, 2020 - WHO announces a name for the new coronavirus disease: COVID-19. - February 19th, 2020 - The U.S. has its first outbreak in a Seattle nursing home which were complicated with loss of lives.. - March 11th, 2020 - WHO declares the virus a pandemic and in less than three months, from the time when this virus was first detected, the virus has spread across the entire planet with cases identified in every country including Greenland. - March 21st, 2020 - Emerging Infectious Disease estimates the risk for death in Wuhan reached values as high as 12% in the epicenter of the epidemic and ≈1% in other, more mildly affected areas. The elevated death risk estimates are probably associated with a breakdown of the healthcare system, indicating that enhanced public health interventions, including social distancing and movement restrictions, should be implemented to bring the COVID-19 epidemic under control." March 21st 2020 -Much of the United States is currently under some form of self- or mandatory quarantine as testing abilities ramp up.. March 24th, 2020 - Hot spots are evolving and identified, particularly in the areas of New York-New Jersey, Washington, and California. Immediate attention is turned to testing, diagnosis, epidemiological containment, clinical trials for drug testing started, and work on a long-term vaccine started. The recovering patients are presenting with mild to severe lung impairment as a result of the viral attack on the alveolar and lung tissues. Clinically significant impairment of pulmonary function appears to be a permanent finding as a direct result of the interstitial lung damage and inflammatory changes that accompanied. This Phase 0, first-in-kind for humans, is use of autologous, cellular stromal vascular fraction (cSVF) deployed intravenously to examine the anti-inflammatory and structural potential to improve the residual, permanent damaged alveolar tissues of the lungs.

NCT ID: NCT03887169 Completed - Clinical trials for Pulmonary Alveolar Proteinosis

Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.

MetPAP
Start date: September 16, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to determine the safety and tolerance of an oral administration of methionine in the treatment of pulmonary alveolar proteinosis due to the double mutation Ala393Thr / Ser567Leu in the MARS gene. This disease is very severe and especially leads to chronic respiratory insufficiency. There is no curative treatment for this disease. The MARS gene encodes the methionine tRNA synthetase (MetRS). Mutations in this gene leads to a defect in MetRS function. In cultured mutated yeast, addition of methionine in culture medium restores MetRS function. Therefore, the investigators hypothesized that treatment of patients with methionine could have beneficial effects on the disease.

NCT ID: NCT03531996 Completed - Clinical trials for Autoimmune Pulmonary Alveolar Proteinosis

The Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis

LongPAP
Start date: April 19, 2018
Phase:
Study type: Observational

The major goal of this study is to conduct a prospective, longitudinal study of autoimmune PAP to examine outcome measures for disease severity of potential use in clinical practice and/or clinical research studies. These results will impact the field by: 1) improving an understanding of the clinical course of autoimmune PAP, 2) providing information on various clinical outcome and quality of life outcome measures to guide patients and physicians in making treatment choices, and 3) facilitate the development of pharmaco-therapeutics for autoimmune PAP and 4) better informing PAP researchers.