Pulmonary Alveolar Proteinosis Clinical Trials

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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 18
Healthy Volunteers: f
View:

• 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.

Locations
Other Locations
Germany
Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik
RECRUITING
München
Contact Information
Primary
Yasmine Wasfi, MD, PhD,
yasmine.wasfi@savarapharma.com
1 512 851 1364
Backup
Brian Maurer
brian.mauer@savarapharma.com
1 512 774 5786
Time Frame
Start Date: 2025-10-22
Estimated Completion Date: 2027-12
Participants
Target number of participants: 5
Treatments
Experimental: molgramostim
Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.
Related Therapeutic Areas
Sponsors
Leads: Savara Inc.

This content was sourced from clinicaltrials.gov