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

Savara

Status and phase

Not yet enrolling

Phase 3

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Treatments

Drug: Molgramostim

Study type

Interventional

Funder types

Industry

Identifiers

NCT06431776

SAV006-04

Details and patient eligibility

About

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.

Full description

This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).

The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.

The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.

Enrollment

5 estimated patients

Sex

All

Ages

6 to 18 years old

Volunteers

No Healthy Volunteers

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