Clinical trials: Proteinosis
Pulmonary Alveolar Proteinosis, Mutation Ala393Thr of the MARS Gene
Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.
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.
- Conditions
- Pulmonary Alveolar Proteinosis, Mutation Ala393Thr of the MARS Gene, mutationSer567Leu of the MARS Gene
- Trial name
- MetPAP (NCT03887169)
- Testing
- Methionine, Vitamin B12, B9, B6, C supplementation, Methionine/homocysteine Dosage
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pulmonary Alveolar Proteinosis
Evaluation and Treatment Planning of Patients With PAP Using Thrive Ultra Short Echo Time MRI and CT
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…
- Trial name
- PAP (NCT02081092)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Pulmonary Alveolar Proteinosis
Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in…
- Trial name
- PAP (NCT01983657)
- Testing
- rhGM-CSF, Whole Lung Lavage(WLL)
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Shanghai Pulmonary Hospital, Shanghai, China
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
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 (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, 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
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.
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
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
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