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.
Status | Not yet recruiting |
Enrollment | 5 |
Est. completion date | August 2027 |
Est. primary completion date | January 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years to 18 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
Lead Sponsor | Collaborator |
---|---|
Savara Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Adverse Events | Adverse events (AEs), including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain). | 48 weeks | |
Other | Anti-GM-CSF Ab titer | Titers of anti-GM-CSF antibodies | 0, 4, 12,24,48 and 52 weeks | |
Other | FEV1 | Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) | 24 and 48-weeks | |
Other | FVC | Change from Baseline in Forced vital capacity (FVC) (% predicted) | 24 and 48-weeks | |
Primary | DLCO | Change in Hb-adjusted % predicted DLCO from Baseline. | 24 weeks | |
Secondary | DLCO | Change in Hb-adjusted % predicted DLCO from Baseline . | 48-weeks | |
Secondary | 6-minute walk distance | Absolute change from Baseline in 6-minute walk distance (6MWD) | 24-weeks | |
Secondary | 6-minute walk distance | Absolute change from Baseline in 6-minute walk distance (6MWD). | 48-weeks | |
Secondary | PedsQL | Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score. | 24-weeks | |
Secondary | PedsQL | Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score. | 48-weeks | |
Secondary | Oxygen Saturation (SpO2) | Absolute change from Baseline in oxygen saturation (SpO2) | 24 weeks | |
Secondary | Oxygen Saturation (SpO2) | Absolute change from Baseline in oxygen saturation (SpO2) | 48 weeks |
Status | Clinical Trial | Phase | |
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