Inhaled molgramostim for children with autoimmune pulmonary alveolar proteinosis
An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
This trial will test whether daily inhaled molgramostim can help children and teens (ages 6–18) with autoimmune pulmonary alveolar proteinosis breathe better and be more active.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 5 (estimated) |
| Ages | 6 Years to 18 Years |
| Sex | All |
| Sponsor | Savara Inc. Industry-sponsored |
| Locations | 1 site (München) |
| Trial ID | NCT06431776 on ClinicalTrials.gov |
What this trial studies
This open-label, single-arm Phase 3 trial gives daily nebulized molgramostim to pediatric participants aged 6–18 with confirmed autoimmune PAP for 48 weeks after a 4-week screening period, followed by a 4-week safety follow-up. Diagnosis must be confirmed by a positive anti-GM-CSF autoantibody test plus compatible symptoms, high-resolution chest CT, bronchoalveolar lavage cytology, or lung biopsy. Participants will visit the clinic about every 12 weeks, keep a diary of oxygen use, and undergo lung function and activity assessments; whole-lung lavage is permitted as a rescue therapy for worsening disease. Primary outcomes include changes in breathing tests (including DLCO), activity measures, and overall safety and tolerability.
Who should consider this trial
Good fit: Children and adolescents aged 6 to 18 with confirmed autoimmune PAP by anti-GM-CSF antibody testing, compatible imaging/biopsy/BAL findings, and a DLCO ≤70% predicted at screening are the ideal candidates.
Not a fit: Patients with hereditary or secondary forms of PAP, surfactant metabolic disorders, very mild disease (DLCO >70%), or recent whole-lung lavage are unlikely to benefit from this treatment.
Why it matters
Potential benefit: If successful, inhaled molgramostim could improve lung function and daily activity and reduce the need for whole-lung lavage in children with aPAP.
How similar studies have performed: Inhaled GM-CSF therapies have shown clinical benefit in adults with autoimmune PAP, but pediatric evidence and molgramostim-specific data are limited.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
München
- Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik — München, Germany (Recruiting)
Study contacts
- Principal investigator: Matthias Griese, MD, — Ludwig Maximilians Universität München
- Study coordinator: Yasmine Wasfi, MD, PhD,
- Email: yasmine.wasfi@savarapharma.com
- Phone: 1 512 851 1364
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.