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Clinical Trial Summary

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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06431776
Study type Interventional
Source Savara Inc.
Contact Yasmine Wasfi, MD, PhD,
Phone 1 512 851 1364
Email yasmine.wasfi@savarapharma.com
Status Not yet recruiting
Phase Phase 3
Start date August 2024
Completion date August 2027

See also
  Status Clinical Trial Phase
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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