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

Launched by SAVARA INC. · May 22, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called molgramostim for children and teenagers aged 6 to 18 who have a condition known as autoimmune pulmonary alveolar proteinosis (aPAP). The main goal is to see how well molgramostim helps improve breathing and daily activities, as well as to check how safe it is for young patients. In this study, all participants will receive molgramostim, which they will take once a day using a nebulizer (a device that turns the medicine into a mist for easier breathing) for a full year. Participants will also have check-up visits at the clinic every three months and keep a diary to track any use of oxygen.

To be eligible for this trial, children must be between 6 and 18 years old and have a confirmed diagnosis of aPAP, which can be determined through specific tests like lung biopsies or imaging scans. They must also show certain test results indicating their lung function is affected. However, children with different types of pulmonary alveolar proteinosis or those who have recently undergone specific treatments will not be eligible. This trial is currently not recruiting participants yet, but it represents an important step in finding new treatments for this rare lung condition in young patients.

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