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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06431776
Other study ID # SAV006-04
Secondary ID
Status Not yet recruiting
Phase Phase 3
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
See also
  Status Clinical Trial Phase
Not yet recruiting NCT06111846 - Study of Human Bone Marrow Mesenchymal Stem Cells in aPAP Phase 2
Active, not recruiting NCT04544293 - Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Phase 3
Completed NCT02702180 - Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis Phase 2
Completed NCT00901511 - Inhaled GM-CSF Therapy of Autoimmune PAP Phase 2
Completed NCT03006146 - Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis Phase 1
Recruiting NCT02243228 - Inhalation of Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) for Autoimmune Pulmonary Alveolar Proteinosis (PAP) Phase 2
Completed NCT03231033 - Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis Phase 1
Completed NCT03531996 - The Longitudinal Evaluation of Autoimmune Pulmonary Alveolar Proteinosis
Completed NCT03482752 - Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Phase 3