Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Launched by SAVARA INC. · May 22, 2024

Keywords

ClinConnect Summary

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 e...

Gender

ALL

Eligibility criteria

• 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

Trial Officials