Ignite Creation Date:
2024-06-16 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 3:30 PM
Study NCT ID:
NCT06431776
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-05-29
First Post:
2024-05-22
Brief Title:
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis aPAP
Sponsor:
Savara Inc
Organization:
Savara Inc
Study Overview
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
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed 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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None