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

Phase 3

Not yet recruiting

• Conditions: Autoimmune Pulmonary Alveolar Proteinosis
• Interventions: Drug: Molgramostim

• Registration Number: NCT06431776
• Lead Sponsor: Savara Inc.

Brief 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.

Detailed 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.

Study Timeline

• Status Verified: 2024-05
• Study First Submitted: 2024-05-22
• Study First Submitted QC: 2024-05-22
• Last Update Submitted: 2024-05-22
• Study First Posted: 2024-05-29
• Last Update Posted: 2024-05-29
• Study Start: 2024-08
• Primary Completion: 2027-01
• Study Completion: 2027-08

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 5

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
molgramostim	Molgramostim	Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.

Primary Outcome Measures

Name	Time	Method
DLCO	24 weeks	Change in Hb-adjusted % predicted DLCO from Baseline.

Secondary Outcome Measures

Name	Time	Method
6-minute walk distance	48-weeks	Absolute change from Baseline in 6-minute walk distance (6MWD).
Oxygen Saturation (SpO2)	48 weeks	Absolute change from Baseline in oxygen saturation (SpO2)
DLCO	48-weeks	Change in Hb-adjusted % predicted DLCO from Baseline .
PedsQL	48-weeks	Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.

Trial Locations

Locations (1)

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States