Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06431776 | Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP). | PHASE3 | RECRUITING | 5 | — | — | Oct 22, 2025 | Dec 1, 2027 | Feb 19, 2026 | 1 | Germany |
| NCT04544293 | Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) | PHASE3 | ACTIVE NOT_RECRUITING | 164 | — | — | May 19, 2021 | May 30, 2027 | Aug 7, 2025 | 54 | United States, Australia +16 |
| NCT02702180 | Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis | PHASE2 | COMPLETED | 139 | — | — | Mar 21, 2016 | Sep 27, 2019 | Apr 12, 2023 | 30 | United States, Australia +16 |
Change in Hb-adjusted % predicted DLCO from Baseline.
As a measure of pulmonary gas transfer, a standardized lung function test, DLCO, was conducted. The single-breath DLCO test was performed in accordance with American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines for DLCO testing. Results reported as % predicted DLCO adjusted for hemoglobin concentration (%predicted DLCOadj).
Measurement of (A-a)DO2 was done by blood gas analysis. An arterial blood sample was collected in the supine position, after resting for at least 10 minutes (or longer if required to achieve stable oxygen saturation). The sample was analyzed for arterial oxygen tension (PaO2) and partial pressure of carbon dioxide (PaCO2). The calculation of (A-a)DO2 was done centrally by using a formula described in the protocol.
| Arm | Type | Description |
|---|---|---|
| molgramostim | EXPERIMENTAL | Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks. |
| Placebo | PLACEBO_COMPARATOR | Double-blind treatment with placebo (PBO) nebulizer solution once daily for 48 weeks |
| Molgramostim Open-label Extension | EXPERIMENTAL | Open-label treatment with molgramostim nebulizer solution 300 µg once daily (Mol OD) for 96 weeks |
| Double-blind molgramostim once daily | EXPERIMENTAL | Inhalation of molgramostim nebuliser solution 300 mcg once daily for 24 weeks |
| Double-blind molgramostim intermittent | EXPERIMENTAL | Inhalation of molgramostim nebuliser solution 300 mcg for 7 days and placebo nebuliser solution for 7 days for 24 weeks (12 cycles) |
| Double-blind placebo | PLACEBO_COMPARATOR | Inhalation of placebo nebuliser solution once daily for 24 weeks |
| Open-label molgramostim intermittent | EXPERIMENTAL | Inhalation of molgramostim nebuliser solution 300 mcg for 7 days and placebo nebuliser solution for 7 days for 24 or 48 weeks from completion of the double-blind period |
| Name | Type | Description |
|---|---|---|
| Molgramostim | DRUG | Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds. |
| Placebo | DRUG | Matching placebo nebulizer solution |
| Molgramostim Open-label | DRUG | Molgramostim 300 µg nebulizer solution |
| PARI eFlow nebulizer system | DEVICE | PARI eFlow nebulizer system |
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 t...