Molgramostim for Autoimmune pulmonary alveolar proteinosis | Pulmonary alveolar proteinosis

Inclusion criteria

• {"criterion_text":"- Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable).\n- 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.\n- Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP prior to screening.\n- Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening."}

Exclusion criteria

• {"criterion_text":"- Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.\n- Have undergone treatment with WLL within 1 month of Baseline."}

Primary endpoints

• {"endpoint_text":"- Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.","definition_or_measurement_approach":"Change from Baseline to Week 24 measured as hemoglobin-adjusted percent predicted diffusing capacity of the lung for carbon monoxide (DLCO)."}

Secondary endpoints

• {"endpoint_text":"- Change in Hb-adjusted % predicted DLCO from Baseline to Week 48.","definition_or_measurement_approach":"Change from Baseline to Week 48 measured as hemoglobin-adjusted % predicted DLCO."}
• {"endpoint_text":"- Absolute change from Baseline in oxygen saturation (SpO2) at rest after 24 and 48 weeks of treatment.","definition_or_measurement_approach":"Absolute change from Baseline in resting oxygen saturation (SpO2) measured at Weeks 24 and 48."}
• {"endpoint_text":"- Absolute change from Baseline in 6-minute walk distance (6MWD) after 24 and 48 weeks of treatment.","definition_or_measurement_approach":"Absolute change from Baseline in distance walked in 6 minutes (meters) measured at Weeks 24 and 48."}
• {"endpoint_text":"- Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score after 24 and 48 weeks of treatment.","definition_or_measurement_approach":"Change from Baseline in PedsQL Generic Core Scale score measured at Weeks 24 and 48."}
• {"endpoint_text":"- AEs, including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain).","definition_or_measurement_approach":"Safety assessments including adverse event reporting, pulmonary function test abnormalities, laboratory safety results, and pre-specified AESIs (hypersensitivity, chest pain)."}
• {"endpoint_text":"- Titers of anti-GM-CSF antibodies at Baseline and after 4, 12, 24, 48, and 52 weeks.","definition_or_measurement_approach":"Measurement of anti-GM-CSF antibody titers at specified timepoints (Baseline, Weeks 4, 12, 24, 48, 52)."}
• {"endpoint_text":"- Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) and forced vital capacity (FVC) (% predicted) after 24 and 48 weeks of treatment.","definition_or_measurement_approach":"Change from Baseline in FEV1 and FVC expressed as percent predicted, measured at Weeks 24 and 48."}

Germany

Earliest CTIS Part Ii Submission Date

31-03-2025

Latest Decision Or Authorization Date

02-02-2026

Processing Time Days

307

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Savara ApS

Organisation Type

Pharmaceutical company

Country Of Registered Address

Denmark

Contract research organisations

Name

Parexel International (IRL) Limited

Responsibilities

sponsorDuties codes: [1,10,11,12,13,5,6,7,8]; general CRO services (contact: Clinicaltrial.Enquiries@parexel.com)

Name

Klifo A/S

Responsibilities

Secondary packaging, IMP CT supply (contact: Jette.Andersen@klifo.com)

Name

BioAgilytix Europe GmbH

Responsibilities

anti-GM-CSF analysis (contact: jessica.gaedicke@bioagilytix.de)

Third parties

• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: [1,10,11,12,13,5,6,7,8]","organisation_type":"Pharmaceutical company"}
• {"country":"Denmark","full_name":"Klifo A/S","duties_or_roles":"Secondary packaging, IMP CT supply","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"BioAgilytix Europe GmbH","duties_or_roles":"anti-GM-CSF analysis","organisation_type":"Pharmaceutical company"}