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

NCT ID: NCT06431776

Last Updated: 2024-05-29

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-08-31
• Study Completion Date: 2027-08-31

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.

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

molgramostim

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

Group Type: EXPERIMENTAL

Molgramostim

Intervention Type: DRUG

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

Interventions

Molgramostim

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

Intervention Type: DRUG

Other Intervention Names

Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF).

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Savara Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Yasmine Wasfi, MD, Ph.D.

Role: STUDY_DIRECTOR

Savara Inc.

Locations

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Central Contacts

Yasmine Wasfi, MD, PhD,

Role: CONTACT

yasmine.wasfi@savarapharma.com

1 512 851 1364

Raymond D Pratt, MD

Role: CONTACT

ray.pratt@savarapharma.com

1 240 899 7058

Other Identifiers

SAV006-04

Identifier Type: -

Identifier Source: org_study_id