Autoimmune Pulmonary Alveolar Proteinosis Clinical Trial
Official title:
An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of 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. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP. This is an open-label study: all participants will receive treatment with molgramostim. Patients will: - Take molgramostim once daily via nebulizer every day for 12 months. - Visit the clinic approximately every 12 weeks for checkups and tests. - Keep a diary of any oxygen use.
This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP). The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology. The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Not yet recruiting |
NCT06111846 -
Study of Human Bone Marrow Mesenchymal Stem Cells in aPAP
|
Phase 2 | |
Active, not recruiting |
NCT04544293 -
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
|
Phase 3 | |
Completed |
NCT02702180 -
Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis
|
Phase 2 | |
Completed |
NCT00901511 -
Inhaled GM-CSF Therapy of Autoimmune PAP
|
Phase 2 | |
Completed |
NCT03006146 -
Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
|
Phase 1 | |
Recruiting |
NCT02243228 -
Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP)
|
Phase 2 | |
Completed |
NCT03231033 -
Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis
|
Phase 1 | |
Completed |
NCT03531996 -
The Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis
|
||
Completed |
NCT03482752 -
Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
|
Phase 3 |