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
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
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(PAP)
The Study of Mesenchymal Stem Cells Treat Autoimmune Pulmonary Alveolar Proteinosis in Vitro
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.
- Trial name
- NCT05640687
- Where
- China
- Volunteers
- 26 people
- Sponsor
- Guangzhou Institute of Respiratory Disease
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
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
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
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 (PAP), Severe Mucocutaneous Candidiasis
Rituximab for Anti-cytokine Autoantibody-Associated Diseases
Background: Healthy people have white blood cells that protect them against bacteria, viruses, and fungi. However, some people have diseases which cause the body to make white blood cells that do not work properly.
- Trial name
- NCT01842386
- Testing
- Rituximab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
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
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
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, 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, 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
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.
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
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