Clinical trials: Autoimmune Pulmonary Alveolar 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
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, 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, 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
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
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.
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
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.
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
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.
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
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.
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
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)
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
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.