Clinical trials: Pulmonary Alveolar Proteinosis
Hereditary Pulmonary Alveolar Proteinosis
Safety and Efficacy of PMT Therapy of hPAP
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…
- Trial name
- NCT05761899
- Testing
- Gene-Corrected Macrophages administered by bronchoscopic instillation
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Pulmonary Alveolar Proteinosis
Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
This study aims to explore a new therapeutic approach: the feasibility, safety and preliminary efficacy of directly spraying GM-CSF into the airway through bronchoscopy for the treatment of aPAP.
- Trial name
- aPAP and GMCSF (NCT06989333)
- Testing
- GM-CSF (granulocyte-macrophage colony-stimulating factor)
- Volunteers
- 10 people
- Sponsor
- Jiuwu Bai
Pulmonary Alveolar Proteinosis
Updated Severity and Prognosis Score of Pulmonary Alveolar Proteinosis
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.
- Trial name
- NCT04516577
- Testing
- Assesssing the severity of pulmonary alveolar proteinosis
- Where
- China
- Volunteers
- 231 people
- Sponsor
- Shanghai Pulmonary Hospital, Shanghai, China
Pulmonary Alveolar Proteinosis (PAP), MARS
Macrophages, GM-CSF and MARS Proteinosis
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.
- Conditions
- Pulmonary Alveolar Proteinosis (PAP), MARS, Genetic Mutation
- Trial name
- MacroMARS (NCT04811274)
- Testing
- Blood collection, Bronchoalveolar lavage fluid collection
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Autoimmune Pulmonary Alveolar Proteinosis
Molgramostim Nebulizer Solution Expanded Access Program Protocol
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disorder in which a material called surfactant builds up in the lungs and makes it hard to breathe.
- Trial name
- NCT06546098
- Testing
- Molgramostim nebulizer solution
- Where
- 12 sites in United States
- Sponsor
- Savara Inc.
Hereditary Pulmonary Alveolar Proteinosis
Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)
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.
- Trial name
- FAMPAP (NCT01511068)
- Testing
- Leukine
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Pulmonary Alveolar Proteinosis
Prevalence of Adenosine Deaminase (ADA) Enzyme Deficiency Disease in Adult Patients With Pulmonary Alveolar Proteinosis
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…
- Trial name
- NCT05300360
- Testing
- adenosine deaminase test
- Where
- Turkey (Türkiye)
- Volunteers
- 15 people
- Sponsor
- TRPHARM
Pulmonary Alveolar Proteinosis, Autoimmune
SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study
Objective: Evaluate Pharmacokinetics and determine the safety of GM-CSF single dose inhalation. Study Design: Pharmacokinetic open study
- Trial name
- NCT02840708
- Testing
- Sargramostim
- Where
- Japan
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Niigata University Medical & Dental Hospital
Pulmonary Alveolar Proteinosis, Treatment
Sequential Therapy With WLL/Inhaling GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis
The purpose of the study is to evaluate the effectiveness of the sequential therapy with whole lung Lavage (WLL)/inhaling granulocyte-macrophage colony stimulating factor, compared to WLL only, for adult patients with severe…
- Trial name
- NCT03316651
- Testing
- GM-CSF
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Dai Huaping
Autoimmune Pulmonary Alveolar Proteinosis
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim (MOL) or placebo (PBO) for 48 weeks.
- Trial name
- IMPALA-2 (NCT04544293)
- Testing
- Molgramostim, Molgramostim Open-label
- Where
- 54 sites in United States, Australia and 16 more
- Volunteers
- 164 people
- Sponsor
- Savara Inc.
Pulmonary Alveolar Proteinosis
GM-CSF in Patients With Pulmonary Alveolar Proteinosis
This is a study to determine the efficacy and safety of granulocyte-macrophage colony-stimulating factor (GM-CSF, sargramostim) administered subcutaneously to patients with pulmonary alveolar proteinosis (PAP).
- Trial name
- NCT00030056
- Testing
- GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim)
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- The Cleveland Clinic
Pulmonary Alveolar Proteinosis, Bronchiectasis
Inhaled Molgramostim (rhGM-CSF) in Healthy Adult Subjects
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.
- Conditions
- Pulmonary Alveolar Proteinosis, Bronchiectasis, Cystic Fibrosis, Acute Respiratory Distress Syndrome
- Trial name
- NCT02468908
- Testing
- Molgramostim
- Where
- United Kingdom
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Savara Inc.
Autoimmune Pulmonary Alveolar Proteinosis
Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis
This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor [rhGM-CSF]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients.
- Trial name
- IMPALA (NCT02702180)
- Testing
- Molgramostim, PARI eFlow nebulizer system
- Where
- 30 sites in United States, Australia and 16 more
- Volunteers
- 139 people
- Sponsor
- Savara Inc.