Ignite Creation Date:
2024-10-26 @ 3:37 PM
Last Modification Date:
2024-10-26 @ 3:37 PM
Study NCT ID:
NCT06546098
Status:
AVAILABLE
Last Update Posted:
None
First Post:
2024-08-05
Brief Title:
Molgramostim Nebulizer Solution Expanded Access Program Protocol
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Expanded Access to Inhaled Molgramostim Nebulizer Solution in Adults with Autoimmune Pulmonary Alveolar Proteinosis aPAP
Status:
AVAILABLE
Status Verified Date:
2024-10
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:
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 In addition to shortness of breath people with aPAP can experience persistent cough overwhelming fatigue unintentional changes in weight chest or back pain suddenly feeling out of shape and general discomfort
Currently there are no approved medications for aPAP in the United States but the symptoms of aPAP can be treated with whole lung lavage WLL WLL is an invasive procedure that temporarily removes surfactant and it can result in serious consequences like trauma to the lung a collapsed lung and prolonged requirement for artificial ventilation
Savara is studying an investigational drug called molgramostim nebulizer solution to see if it activates the cells that help clear surfactant from the lungs which improves oxygen transfer from the lungs to the bloodstream Molgramostim nebulizer solution is administered by inhalation using a hand-held nebulizer In clinical trials molgramostim nebulizer solution has shown improvements in gas exchange and patient reported outcomes
This expanded access program will make molgramostim nebulizer solution available to adult patients with diagnosed aPAP Access must be obtained through the treating physician Patients will dose molgramostim nebulizer solution 300 micrograms mcg once daily and be followed by their physician every 3 months to assess their clinical status and report any adverse events
Currently there are no approved medications for aPAP in the United States but the symptoms of aPAP can be treated with whole lung lavage WLL WLL is an invasive procedure that temporarily removes surfactant and it can result in serious consequences like trauma to the lung a collapsed lung and prolonged requirement for artificial ventilation
Savara is studying an investigational drug called molgramostim nebulizer solution to see if it activates the cells that help clear surfactant from the lungs which improves oxygen transfer from the lungs to the bloodstream Molgramostim nebulizer solution is administered by inhalation using a hand-held nebulizer In clinical trials molgramostim nebulizer solution has shown improvements in gas exchange and patient reported outcomes
This expanded access program will make molgramostim nebulizer solution available to adult patients with diagnosed aPAP Access must be obtained through the treating physician Patients will dose molgramostim nebulizer solution 300 micrograms mcg once daily and be followed by their physician every 3 months to assess their clinical status and report any adverse events
Detailed Description:
Autoimmune pulmonary alveolar proteinosis aPAP is a rare disease mediated by autoantibodies targeting granulocyte macrophage colony-stimulating factor GM-CSF resulting in malfunctioning macrophages with impaired surfactant catabolism The latter causes accumulation of surfactant in the alveoli which has a negative impact on gas exchange between lung and blood The clinical course of autoimmune PAP varies among patients with continuously slowly progressive disease in most patients spontaneous improvement in a small percentage 5-7 and rapid progression andor pulmonary fibrosis respiratory failure and death in others The most common cause of death is respiratory failure followed by secondary pulmonary bacterial infections Estimated 5-year mortality rates vary between 10-30 with overall disease-specific survival rates at 5 years exceeding 80
There are currently no approved pharmacological treatments for aPAP in most of the world Whole lung lavage WLL is the primary treatment option currently available for most aPAP patients However its invasive nature limited access and the variable effect of WLLs due to lack of standardization emphasize that there is an unmet need for a non-invasive safe and well-tolerated easily accessible and effective treatment for aPAP patients
The rationale for treating aPAP patients with molgramostim nebulizer solution is based on the capacity of GM-CSF to promote differentiation and mobilization of different myeloid leukocyte subsets including neutrophils tissue macrophagesdendritic cells or their circulating precursors It is crucially involved in anti-microbial pulmonary host defense and ameliorates lung injury by increasing the size and activation of the alveolar macrophage pool GM-CSF also contributes to the proliferation of megakaryocytic and erythroid progenitors and plays a key role in surfactant homeostasis by maturation of alveolar macrophages
Molgramostim is a non-glycosylated recombinant human granulocyte macrophage colony stimulating factor rhGM-CSF produced by using recombinant DNA technology via a bacterial E coli expression system Molgramostim is formulated in a sterile nebulizer solution molgramostim nebulizer solution which is supplied in vials containing 300 µg of molgramostim in 12 mL solution and is administered by inhalation via an investigational eFlow Nebulizer System PARI Pharma GmbH
Results of a completed randomized placebo-controlled clinical trial support the safety tolerability and efficacy of inhaled molgramostim nebulizer solution as a treatment for autoimmune PAP patients
There are currently no approved pharmacological treatments for aPAP in most of the world Whole lung lavage WLL is the primary treatment option currently available for most aPAP patients However its invasive nature limited access and the variable effect of WLLs due to lack of standardization emphasize that there is an unmet need for a non-invasive safe and well-tolerated easily accessible and effective treatment for aPAP patients
The rationale for treating aPAP patients with molgramostim nebulizer solution is based on the capacity of GM-CSF to promote differentiation and mobilization of different myeloid leukocyte subsets including neutrophils tissue macrophagesdendritic cells or their circulating precursors It is crucially involved in anti-microbial pulmonary host defense and ameliorates lung injury by increasing the size and activation of the alveolar macrophage pool GM-CSF also contributes to the proliferation of megakaryocytic and erythroid progenitors and plays a key role in surfactant homeostasis by maturation of alveolar macrophages
Molgramostim is a non-glycosylated recombinant human granulocyte macrophage colony stimulating factor rhGM-CSF produced by using recombinant DNA technology via a bacterial E coli expression system Molgramostim is formulated in a sterile nebulizer solution molgramostim nebulizer solution which is supplied in vials containing 300 µg of molgramostim in 12 mL solution and is administered by inhalation via an investigational eFlow Nebulizer System PARI Pharma GmbH
Results of a completed randomized placebo-controlled clinical trial support the safety tolerability and efficacy of inhaled molgramostim nebulizer solution as a treatment for autoimmune PAP patients
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: