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Recruiting NCT06431776

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

Phase III Interventional Autoimmune Pulmonary Alveolar Proteinosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Molgramostim.
Who it may be relevant to
Registry conditions: Autoimmune Pulmonary Alveolar Proteinosis. Basic parameters: 6 years — 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Germany
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Overview

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.

Interventions

  • Drug Molgramostim
    Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.

Primary outcome measures

  • DLCO [Time frame: 24 weeks]
Secondary outcome measures (7)
  • DLCO [Time frame: 48-weeks]
  • 6-minute walk distance [Time frame: 24-weeks]
  • 6-minute walk distance [Time frame: 48-weeks]
  • PedsQL [Time frame: 24-weeks]
  • PedsQL [Time frame: 48-weeks]
  • Oxygen Saturation (SpO2) [Time frame: 24 weeks]
  • Oxygen Saturation (SpO2) [Time frame: 48 weeks]

Eligibility 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Germany · 1 center
  • Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik u — München

Identifiers

NCT: NCT06431776 · SAV006-04

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗