Inhaled Molgramostim in Pediatric Participants With Autoi... | NCT06431776

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

NCT06431776

Sponsor

Savara Inc.

Status

Recruiting

Last Update Posted

Feb 19, 2026Actual

Enrollment

5Estimated

Phase

Phase 3

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Interventions

Molgramostim

Countries

Germany

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Oct 22, 2025Actual

Primary Completion Date

Jun 2027Estimated

Completion Date

Dec 2027Estimated

First Submitted Date

May 22, 2024

First Submission Date that Met QC Criteria

May 22, 2024

First Posted Date

May 29, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 17, 2026

Last Update Posted Date

Feb 19, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

5Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
molgramostim	Experimental	Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.	Drug: Molgramostim

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Molgramostim	Drug	Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
DLCO	Change in Hb-adjusted % predicted DLCO from Baseline.	24 weeks

Secondary Outcomes

Measure	Description	Time Frame
DLCO	Change in Hb-adjusted % predicted DLCO from Baseline .	48-weeks
6-minute walk distance	Absolute change from Baseline in 6-minute walk distance (6MWD)

Other Outcomes

Measure	Description	Time Frame
Adverse Events	Adverse events (AEs), including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain).	48 weeks
Anti-GM-CSF Ab titer

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Yasmine Wasfi, MD, PhD,	Contact	1 512 851 1364		yasmine.wasfi@savarapharma.com
Brian Maurer	Contact	1 512 774 5786		brian.mauer@savarapharma.com

Overall Officials

Name	Affiliation	Role
Matthias Griese, MD,	Ludwig Maximilians Universität München	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik	Recruiting	München		80337	Germany

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
C567049	Pulmonary Alveolar Proteinosis, Acquired
D011649	Pulmonary Alveolar Proteinosis

Ancestor Terms

ID	Term
D008171	Lung Diseases
D012140	Respiratory Tract Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C082430	molgramostim
D003115	Colony-Stimulating Factors
C082856	regramostim

Ancestor Terms

ID	Term
D006023	Glycoproteins
D006001	Glycoconjugates
D002241	Carbohydrates
D016298	Hematopoietic Cell Growth Factors

Browse Leaves

Not provided

Browse Branches

Not provided