Autoimmune Pulmonary Alveolar Proteinosis
Conditions
Keywords
alveolar proteinosis, autoimmune, lung lavage, GM-CSF, children
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.
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.
Interventions
DRUGMolgramostim
Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.
Sponsors
Savara Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label study
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Be ≥6 and \<18 years of age, at the time of signing the informed consent and informed assent (if applicable). * Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest. * Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP. * Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.
Exclusion criteria
* Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production. * Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| DLCO | 24 weeks | Change in Hb-adjusted % predicted DLCO from Baseline. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| DLCO | 48-weeks | Change in Hb-adjusted % predicted DLCO from Baseline . |
| 6-minute walk distance | 24-weeks | Absolute change from Baseline in 6-minute walk distance (6MWD) |
| PedsQL | 24-weeks | Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score. |
| Oxygen Saturation (SpO2) | 24 weeks | Absolute change from Baseline in oxygen saturation (SpO2) |
Countries
Germany
Contacts
CONTACTYasmine Wasfi, MD, PhD,
CONTACTBrian Maurer
PRINCIPAL_INVESTIGATORMatthias Griese, MD,
Ludwig Maximilians Universität München
Outcome results
None listed