NCT06431776

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for phase_3

Timeline
19mo left

Started Oct 2025

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress26%
Oct 2025Dec 2027

First Submitted

Initial submission to the registry

May 22, 2024

Completed
7 days until next milestone

First Posted

Study publicly available on registry

May 29, 2024

Completed
1.4 years until next milestone

Study Start

First participant enrolled

October 22, 2025

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2027

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Last Updated

February 19, 2026

Status Verified

February 1, 2026

Enrollment Period

1.6 years

First QC Date

May 22, 2024

Last Update Submit

February 17, 2026

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Keywords

alveolar proteinosisautoimmunelung lavageGM-CSFchildren

Outcome Measures

Primary Outcomes (1)

  • DLCO

    Change in Hb-adjusted % predicted DLCO from Baseline.

    24 weeks

Secondary Outcomes (7)

  • DLCO

    48-weeks

  • 6-minute walk distance

    24-weeks

  • 6-minute walk distance

    48-weeks

  • PedsQL

    24-weeks

  • PedsQL

    48-weeks

  • +2 more secondary outcomes

Other Outcomes (4)

  • Adverse Events

    48 weeks

  • Anti-GM-CSF Ab titer

    0, 4, 12,24,48 and 52 weeks

  • FEV1

    24 and 48-weeks

  • +1 more other outcomes

Study Arms (1)

molgramostim

EXPERIMENTAL

Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.

Drug: Molgramostim

Interventions

MolgramostimDRUG

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

Also known as: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF).
molgramostim

Eligibility Criteria

Age6 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik

München, 80337, Germany

RECRUITING

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis, AcquiredPulmonary Alveolar Proteinosis

Interventions

molgramostimColony-Stimulating Factorsregramostim

Condition Hierarchy (Ancestors)

Lung DiseasesRespiratory Tract Diseases

Intervention Hierarchy (Ancestors)

GlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Officials

  • Matthias Griese, MD,

    Ludwig Maximilians Universität München

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Yasmine Wasfi, MD, PhD,

CONTACT

1 512 851 1364yasmine.wasfi@savarapharma.com

Brian Maurer

CONTACT

1 512 774 5786brian.mauer@savarapharma.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Open-label study
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 22, 2024

First Posted

May 29, 2024

Study Start

October 22, 2025

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

December 1, 2027

Last Updated

February 19, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

DE(1)