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

Autoimmune Pulmonary Alveolar Proteinosis Clinical Trial

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).

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06431776
• Other study ID #: SAV006-04
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: August 2024
• Est. completion date: August 2027

Study information

• Verified date: May 2024
• Source: Savara Inc.
• Contact: Yasmine Wasfi, MD, PhD,
• Phone: 1 512 851 1364
• Email: yasmine.wasfi[@]savarapharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 5
• Est. completion date: August 2027
• Est. primary completion date: January 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Autoimmune Diseases
• Autoimmune Pulmonary Alveolar Proteinosis
• Pulmonary Alveolar Proteinosis

Intervention

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

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Savara Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	Anti-GM-CSF Ab titer	Titers of anti-GM-CSF antibodies	0, 4, 12,24,48 and 52 weeks
Other	FEV1	Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted)	24 and 48-weeks
Other	FVC	Change from Baseline in Forced vital capacity (FVC) (% predicted)	24 and 48-weeks
Primary	DLCO	Change in Hb-adjusted % predicted DLCO from Baseline.	24 weeks
Secondary	DLCO	Change in Hb-adjusted % predicted DLCO from Baseline .	48-weeks
Secondary	6-minute walk distance	Absolute change from Baseline in 6-minute walk distance (6MWD)	24-weeks
Secondary	6-minute walk distance	Absolute change from Baseline in 6-minute walk distance (6MWD).	48-weeks
Secondary	PedsQL	Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.	24-weeks
Secondary	PedsQL	Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.	48-weeks
Secondary	Oxygen Saturation (SpO2)	Absolute change from Baseline in oxygen saturation (SpO2)	24 weeks
Secondary	Oxygen Saturation (SpO2)	Absolute change from Baseline in oxygen saturation (SpO2)	48 weeks