Clinical trials: Proteinosis
Autoimmune Pulmonary Alveolar Proteinosis
Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
SAV006-03 is an open-label extension study for participants who had completed the IMPALA study. At the baseline visit, eligible participants may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human…
- Trial name
- IMPALA-X (NCT03482752)
- Testing
- Molgramostim
- Where
- 13 sites in Denmark, France and 8 more
- Volunteers
- 60 people
- Sponsor
- Savara Inc.
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
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, Autoimmune
Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
Objective: Determine the safety and efficacy of GM-CSF inhalation in patients with aPAP. Study Design: multi-center, randomized, double-blind, placebo- controlled, safety/efficacy study.
- Trial name
- NCT02835742
- Testing
- Sargramostim
- Where
- Japan
- Volunteers
- 78 people
- Sponsor
- Niigata University Medical & Dental Hospital
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.
Autoimmune Pulmonary Alveolar Proteinosis
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18.
- Trial name
- NCT06431776
- Testing
- Molgramostim
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- Savara Inc.
Autoimmune Pulmonary Alveolar Proteinosis
Study of Human Bone Marrow Mesenchymal Stem Cells in APAP
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.
- Trial name
- NCT06111846
- Testing
- human Bone Marrow Mesenchymal Stem Cell (hBMMSC)
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
Autoimmune Pulmonary Alveolar Proteinosis
Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis
Pulmonary alveolar proteinosis (PAP) is a syndrome of surfactant accumulation, respiratory failure, and innate immune deficiency for which therapy remains limited to whole lung lavage (WLL), an invasive physical procedure to…
- Trial name
- PioPAP (NCT03231033)
- Testing
- Pioglitazone
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Pulmonary Alveolar Proteinosis
A National Registry For Pulmonary Alveolar Proteinosis
The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the diagnosis of PAP, increase awareness and knowledge of PAP, and give…
- Trial name
- NCT02461615
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Proteinosis, Gallbladder Carcinoma
A Study of Circulating Exosome Proteomics In Gallbladder Carcinoma Patients
This research will be the first study for exosome purified from blood in gallbladder carcinoma patients. Proteomics studies will be done in both tumor tissue and the circulating exosome from blood specimens.
- Trial name
- EXOGBC001 (NCT03581435)
- Where
- China
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- liu yingbin
Autoimmune Pulmonary Alveolar Proteinosis
Inhaled GM-CSF Therapy of Autoimmune PAP
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.
- Trial name
- NCT00901511
- Testing
- Baseline WLL, Sargramostim, Rescue WLL
- Where
- Italy
- Volunteers
- 18 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
Pulmonary Alveolar Proteinosis, COPD
Use of cSVF Via IV Deployment for Residual Lung Damage After Symptomatic COVID-19 Infection
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.
- Conditions
- Pulmonary Alveolar Proteinosis, COPD, Idiopathic Pulmonary Fibrosis, Viral Pneumonia
- Trial name
- GARM-COVID19 (NCT04326036)
- Testing
- Microcannula Harvest Adipose Derived tissue stromal vascular fraction (tSVF), Centricyte 1000, IV Deployment Of cSVF In Sterile Normal Saline IV Solution
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Black Tie Medical, 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
Heat Stroke, Early Waking
Study on the Establishment of a System for Early Warning and Prognostic Evaluation of Patients With Heat Stroke
Heat stroke is a clinical syndrome with high incidence and high fatality rate in summer. Patients with liver, kidney, and brain damage are prone to secondary MODS, and the prognosis is poor due to high medical costs.
- Conditions
- Heat Stroke, Early Waking, MODS, Proteinosis
- Trial name
- NCT05155358
- Testing
- Peripheral blood test
- Where
- China
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Xijing Hospital
Pulmonary Alveolar Proteinosis
Multicenter International Cross-Sectional Evaluation of Pulmonary Alveolar Proteinosis Trial
The purpose of this study is to (1) compare a technically improved assay with an existing assay used to measure serum anti-GM-CSF antibodies in stored serum samples previously obtained from patients diagnosed with either primary…
- Trial name
- NCT03007134
- Testing
- Retrospective Chart Review
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 73 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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.
Lung Diseases, Interstitial, Pulmonary Alveolar Proteinosis
European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank
Generation of a common European database and biobank Continous assessment and implementation of guidelines and treatment protocols Establishment of a large observational cohort of chILD patients Determination the value of…
- Conditions
- Lung Diseases, Interstitial, Pulmonary Alveolar Proteinosis, Pulmonary Eosinophilia, Pulmonary Fibrosis
- Trial name
- chILD-EU (NCT02852928)
- Where
- 3 sites in Germany, Turkey (Türkiye) and 1 more
- Volunteers
- 1,000 people
- Sponsor
- Matthias Griese