An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Subjects with Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Conditions

Autoimmune pulmonary alveolar proteinosis

Brief summary

Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.

Detailed description

Change in Hb-adjusted % predicted DLCO from Baseline to Week 48., Absolute change from Baseline in oxygen saturation (SpO2) at rest after 24 and 48 weeks of treatment., Absolute change from Baseline in 6-minute walk distance (6MWD) after 24 and 48 weeks of treatment., Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score after 24 and 48 weeks of treatment., AEs, including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain)., Titers of anti-GM-CSF antibodies at Baseline and after 4, 12, 24, 48, and 52 weeks., Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) and forced vital capacity (FVC) (% predicted) after 24 and 48 weeks of treatment.

Related papers

No related papers found yet.

Interventions

DRUGMolgradex

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.	—

Secondary

Measure	Time frame
Change in Hb-adjusted % predicted DLCO from Baseline to Week 48., Absolute change from Baseline in oxygen saturation (SpO2) at rest after 24 and 48 weeks of treatment., Absolute change from Baseline in 6-minute walk distance (6MWD) after 24 and 48 weeks of treatment., Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score after 24 and 48 weeks of treatment., AEs, including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain)., Titers of anti-GM-CSF antibodies at Baseline and after 4, 12, 24, 48, and 52 weeks., Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) and forced vital capacity (FVC) (% predicted) after 24 and 48 weeks of treatment.	—

Measure

Time frame

Change in Hb-adjusted % predicted DLCO from Baseline to Week 48., Absolute change from Baseline in oxygen saturation (SpO2) at rest after 24 and 48 weeks of treatment., Absolute change from Baseline in 6-minute walk distance (6MWD) after 24 and 48 weeks of treatment., Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score after 24 and 48 weeks of treatment., AEs, including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain)., Titers of anti-GM-CSF antibodies at Baseline and after 4, 12, 24, 48, and 52 weeks., Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) and forced vital capacity (FVC) (% predicted) after 24 and 48 weeks of treatment.

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Countries

Germany

Outcome results

None listed