RecruitingPhase 3

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

Germany5 participantsStarted 2025-10-22

Plain-language 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.

Who can participate

Age range6 Years – 18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

DLCO

Timeframe: 24 weeks

Trial details

NCT IDNCT06431776

SponsorSavara Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-06

Contact for this trial

Yasmine Wasfi, MD, PhD,

1 512 851 1364 yasmine.wasfi@savarapharma.com