Clinical trials: Proteinosis
Autoimmune Pulmonary Alveolar Proteinosis
Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
Autoimmune PAP is a rare lung disease affecting less than 5,000 individuals in US with no FDA-approved pharmacologic therapy. Results from "off-label" use in case reports and clinical studies completed outside of the US indicate…
- Trial name
- NCT03006146
- Testing
- Sargramostim
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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
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
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
Acute Kidney Injury
Etiology, Epidemiology and Prognostics of AKI
To investigate the etiology and epidemiology of acute kidney injury (AKI). To find out risk factors associated with the occurence of AKI.
- Trial name
- NCT02967263
- Where
- China
- Volunteers
- 713 people, healthy volunteers welcome
- Sponsor
- Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
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
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
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, 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
Postoperative Complications, Proteinosis
Preserving Left Colonic Artery During Radical Resection of Rectal Cancer
To evaluate the feasibility and clinical significance of preserving left colonic artery in rectal cancer surgery.The investigators will focus on the effect of preserving left colonic artery during radical resection of rectal…
- Trial name
- NCT03776370
- Testing
- Preserve the left colonic artery, The left colonic artery is not preserved
- Where
- 2 sites in China
- Volunteers
- 200 people
- Sponsor
- Third Military Medical University
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
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
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
Autoimmune Pulmonary Alveolar Proteinosis
Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP)
The purpose of the study is to evaluate if inhaled granulocyte-macrophage colony stimulating factor delay the increase in alveolar-arterial oxygen difference, compared to no treatment, for adult patients with mild-to-moderate…
- Trial name
- NCT02243228
- Testing
- GM-CSF
- Where
- China
- Volunteers
- 42 people
- Sponsor
- Peking Union Medical College Hospital
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
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
Autoimmune Pulmonary Alveolar Proteinosis
The Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis
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.
- Trial name
- LongPAP (NCT03531996)
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Autoimmune Pulmonary Alveolar Proteinosis
A Study of hBMMSC Injection in Patients With aPAP
The purpose of this multicenter, randomized, double-blind, placebo-controlled phase IIb clinical trial is to evaluate the efficacy and safety of intravenous hBMMSC injection in patients with autoimmune pulmonary alveolar…
- Trial name
- NCT07890649
- Testing
- The human Bone Marrow Mesenchymal Stem Cell (hBMMSC) injection, Lactated Ringer's Injection (crystalloid)
- Where
- China
- Volunteers
- 70 people
- Sponsor
- Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
Pulmonary Alveolar Proteinosis, Primary Disease
Prospective Trial of Rituximab for Primary 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.
- Trial name
- PAP (NCT00552461)
- Testing
- rituximab
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- East Carolina University
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.